{"id":98,"date":"2025-07-14T16:15:02","date_gmt":"2025-07-14T16:15:02","guid":{"rendered":"https:\/\/medical.biomarin.com\/en-us\/?page_id=98"},"modified":"2026-08-07T17:44:46","modified_gmt":"2026-08-07T17:44:46","slug":"pipeline-and-clinical-development-programs","status":"publish","type":"page","link":"https:\/\/medical.biomarin.com\/en-us\/pipeline-and-clinical-development-programs\/","title":{"rendered":"Pipeline and Clinical Development Programs"},"content":{"rendered":"<div id=\"acf-block-68752b21849de\" class=\"hero hero-blue hero-video\">\n                            <div class=\"background-video\">\n            <video class=\"background-video-desktop\" src=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/07\/Pipeline_desktop.mp4?v=1.27\" loop autoplay=\"\" playsinline=\"\" muted=\"\" preload=\"\">\n            <\/video>\n            <video class=\"background-video-mobile\" src=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/07\/Pipeline_desktop.mp4?v=1.27\" loop autoplay=\"\" playsinline=\"\" muted=\"\" preload=\"\">\n            <\/video>\n        <\/div>\n                    <a class=\"video-control video-control-play\" href=\"#\">Play<\/a>\n            <a class=\"video-control video-control-pause\" href=\"#\">Pause<\/a>\n            \t<div class=\"overlay\"><\/div>\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t\t<div class=\"hero-content\">\n\t\t\t    \t\t\t\t\t\t\t\t\t\t\t\t    <h1>Pipeline and Clinical Development Programs\n<\/h1>\n\t\t\t\t\t\t\t\t\t\t\t\t    <p>We transform lives through genetic discovery<\/p>\n\t\t\t\t\t\t\t\t\n                <p class=\"inline-buttons\">\n                    \t\t\t\t        <a class=\"button button-arrow\" href=\"https:\/\/clinicaltrials.biomarin.com\/\" target=\"_blank\">Visit the BioMarin Clinical Trials website<\/a>\t\t\t\t                                        <\/p>\n\t\t\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n\n<div id=\"acf-block-68752b218807e\" class=\"vblock pipeline pipeline-listing-wrapper\">\n\t<!-- Pipeline Filter Header\n\tIntegration notes:\n\t- Toggle button-filter-active class to filter button when active\n\t- Component here is generic filter header and is also used for resources (filters themselves differ)\n\t-->\n\t<div class=\"block filter-header\">\n\t\t<div class=\"wrapper\">\n\t\t\t<div class=\"inner-wrapper\">\n\t\t\t\t<h1>Pipeline and Clinical Development Programs<\/h1>\n\t\t\t\t\n\t\t\t\t\t\t\t\t\t<div class=\"filters\">\n\t\t\t\t\t\t<a class=\"button button-filter\" href=\"#\"><span>Search\/Filter<\/span><\/a>\n\t\t\t\t\t\t<div class=\"filters-content\">\n\t\t\t\t\t\t\t<div class=\"wrapper\">\n\t\t\t\t\t\t\t\t<div class=\"inner-wrapper\">\n\t\t\t\t\t\t\t\t\t<form>\n\t\t\t\t\t\t\t\t\t\t<fieldset>\n\t\t\t\t\t\t\t\t\t\t\t<legend>Filter by phase:<\/legend>\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"filter-checkboxes\">\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-phase-observational\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-phase-observational\">Observational<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-phase-1\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-phase-1\">Phase 1<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-phase-2\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-phase-2\">Phase 2<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-phase-3\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-phase-3\">Phase 3<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-phase-4\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-phase-4\">Phase 4<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/fieldset>\n\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<fieldset>\n\t\t\t\t\t\t\t\t\t\t\t\t<legend>Filter by status:<\/legend>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"filter-checkboxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-rec-stat-active-not-recruiting\" value=\"filter-rec-stat-active-not-recruiting\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-rec-stat-active-not-recruiting\">Active, Not Recruiting<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-rec-stat-recruiting\" value=\"filter-rec-stat-recruiting\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-rec-stat-recruiting\">Recruiting<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-rec-stat-enrolling-by-invitation\" value=\"filter-rec-stat-enrolling-by-invitation\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-rec-stat-enrolling-by-invitation\">Enrolling by Invitation<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-rec-stat-not-started\" value=\"filter-rec-stat-not-started\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-rec-stat-not-started\">Not started<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-rec-stat-not-yet-recruiting\" value=\"filter-rec-stat-not-yet-recruiting\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-rec-stat-not-yet-recruiting\">Not yet recruiting<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/fieldset>\n\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t<div class=\"filter-footer\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"filter-footer-reset\">\n\t\t\t\t\t\t\t\t\t\t\t\t<a class=\"button button-text\" href=\"#\">Reset all filters<\/a>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"filter-footer-submit\">\n\t\t\t\t\t\t\t\t\t\t\t\t<a class=\"button button-text\" href=\"#\">Cancel<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"submit\" class=\"button\" value=\"Apply filters\">\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/form>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n\n\t<!-- Research Pipeline block -->\n\t<div class=\"block bg-band pipeline-listing-main\">\n\t\t<div class=\"wrapper\">\n\t\t\t<div class=\"inner-wrapper\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t                    \n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-listing pipeline-skeletal-conditions\">\n\t\t\t\t\t\t<h2 class=\"h3\">Skeletal Conditions<\/h2>\n\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5 trial-phase trial-phase\">Trial phase<\/h3>\n\n\t\t\t\t\t\t\t<div class=\"pipeline-group\" data-trial-phase-class=\"trial-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">Program (Trial)<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">Condition<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">Phase<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-achondroplasia filter-tech-peptide filter-rec-stat-recruiting phase-3\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">BMN 333 Long-Acting CNP<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(333-301 [ASPEN])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Achondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 3<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>A Phase 2\/&#x200B;3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia (ASPEN)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>To evaluate the effects of BMN 333 on growth compared with vosoritide in participants with achondroplasia who have not received any growth-promoting treatments. The study includes 2 parts: the Phase 2 part will select the optimal BMN 333 dose to be used in Phase 3 and determine study continuation into Phase 3; the Phase 3 part will compare the effects of the selected dose of BMN 333 with vosoritide.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Predicted annualized growth velocity (AGV; Phase 2) and AGV (Phase 3)<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Aged &ge; 2 to &lt; 11 years (Phase 2) or &ge; 2 to &lt; 18 years (Phase 3)<\/li>\n<li>Achondroplasia (confirmed by documented genetic testing) and open epiphyses<\/li>\n<li>Tanner Stage I (Phase 2) or any Tanner stage (Phase 3)<\/li>\n<li>Ambulatory and able to stand without assistance<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Change from baseline in standing height, height Z-score, and upper to lower body segment ratio<\/li>\n<li>Incidence of adverse events (AEs), serious AEs, and events of interest<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Any short stature condition other than achondroplasia<\/li>\n<li>History of any renal insufficiency or cardiac\/ cardiovascular disease that places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension<\/li>\n<li>Bone fractures of the long bones or spine within 6 months prior to screening<\/li>\n<li>Used vosoritide, any other approved product (except growth hormone [GH], as detailed below), investigational product, or investigational medical device for the treatment of achondroplasia or short stature at any time<\/li>\n<li>Been treated with GH, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to treatment start<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT07441876\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT07441876<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-achondroplasia filter-tech-observational filter-rec-stat-recruiting phase-4\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Vosoritide<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(111-603 [Acorn])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Achondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 4<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>A multicentre, non-interventional study to evaluate long-term safety in patients with achondroplasia treated with Voxzogo (vosoritide)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This post-authorization safety study will evaluate the long-term safety of treatment with vosoritide in patients with achondroplasia in a real-world setting.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Exposure-adjusted incidence rate of all new bone-related safety events of interest which have not previously been observed in patients<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/catalogues.ema.europa.eu\/node\/3537\/administrative-details\" target=\"_blank\">For reference and comprehensive trial information, visit EMA.Europa.eu EUPAS47514<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-achondroplasia filter-tech-observational filter-rec-stat-recruiting phase-4\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Vosoritide<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(111-605 [VISTA])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Achondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 4<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>A Participant-mediated Observational Virtual Registry of Children With Achondroplasia in the United States (VISTA)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This is an observational study of a pediatric cohort with achondroplasia in the United States. This cohort consists of both individuals treated and untreated with vosoritide.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Height, weight, body mass index, head circumference, annualized grown velocity, surgical procedures and\/or medical interventions by age<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n<h4>Select Inclusion Criteria*<\/h4>\n\n<ul>\n<li class=\"ewa-rteLine\">Physician diagnosis of achondroplasia<\/li>\n<li class=\"ewa-rteLine\">Age at time of enrollment<\/li>\n<li class=\"ewa-rteLine\">Receiving medical care in the United States<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measure*<\/h4>\n<ul>\n<li>Not applicable<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Lack of any medical records<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT06168201\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06168201<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-noonan-syndrome filter-tech-peptide filter-rec-stat-recruiting phase-2\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Vosoritide<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(111-211 [Canopy NS])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Noonan Syndrome<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 2<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>The purpose of this study in children with Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the therapeutic dose will be evaluated up to FAH.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Change from baseline in annualized growth velocity (AGV)<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Patients &ge; 3 years old, and &lt; 10 years old (females) or &lt; 11 years old (males)<\/li>\n<li>Height Z-score &#x2264; -2 standard deviations in reference to the general population of the same age and sex<\/li>\n<li>Have been receiving continuous human growth hormone (hGH) for the treatment of short stature associated with their condition for a minimum of 1 year immediately prior to enrollment<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Patients &ge; 3 years old, and &lt; 11 years old (females) or &lt; 12 years old (males)<\/li>\n<li>Tanner Stage 1<\/li>\n<li>Previous or current hGH treatment for short stature associated with their condition<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Patients with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia<\/li>\n<li>Bone age advanced beyond chronological age by more than 2 years<\/li>\n<li>Evidence of decreased growth velocity (AGV &lt; 1.5 cm\/year) as assessed over a period of at least 6 months and growth plate closure assessed using bilateral lower extremity X-rays<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT06668805\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06668805<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-idiopathic-short-stature filter-tech-peptide filter-rec-stat-recruiting phase-2\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Vosoritide<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(111-210 [Canopy ISS-2])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Idiopathic Short Stature<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 2<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This study will evaluate the effect of multiple doses of vosoritide and the effect of vosoritide compared to human growth hormone (hGH), in children with Idiopathic Short Stature.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Change in annualized growth velocity (AGV), height, and height Z-score<\/li>\n<\/ul>\n<p>&#xA0;<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Patients &ge;3 and &lt;10 (female) or &lt; 11 (males) years of age<\/li>\n<li>Height Z-score &#x2264;-2.25 standard deviations compared to age and sex matched population norms<\/li>\n<li>If &#x2265; 5 years at Screening, must be Tanner Stage I<\/li>\n<\/ul>\n<p>&#xA0;<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Incidence of adverse events<\/li>\n<li>Change in pharmacodynamic parameters (urine cyclic guanine monophosphate, serum collagen X marker)<\/li>\n<li>Pharmacokinetics of vosoritide<\/li>\n<\/ul>\n<p>&#xA0;<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to: Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome), ACAN deficiency<\/li>\n<li>History of treatment with a growth promoting agent<\/li>\n<\/ul>\n<p>&#xA0;<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT06382155\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06382155<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-idiopathic-short-stature filter-tech-observational filter-rec-stat-recruiting observational\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Idiopathic Short Stature<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(111-903 [Canopy ISS-OS])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Idiopathic Short Stature<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Observational<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>A Study to Assess Growth in Children With Idiopathic Short Stature<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This study will collect growth measurements and other variables of interest to generate baseline growth data in children with Idiopathic Short Stature.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Change in growth (annualized growth velocity, height Z-score, height) and anthropometric measures (body mass index [BMI], BMI Z-score)<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Patients &gt; 2 and &le; 14 (female) or &le; 16 (males) years of age<\/li>\n<li>Height Z-score &#x2264;-2.5 standard deviations compared to age and sex matched population norms<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li class=\"ewa-rteLine\">Rates of medical events of interest<\/li>\n<li class=\"ewa-rteLine\">Association between specific variants and growth velocity<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li class=\"ewa-rteLine\">Presence of pituitary hormone deficiencies<\/li>\n<li class=\"ewa-rteLine\">Bone age advanced over chronological age by &gt;3 years<\/li>\n<li class=\"ewa-rteLine\">Received an investigational product or medical device within last 6 months<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT06309979\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06309979<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-hypochondroplasia filter-tech-peptide filter-rec-stat-recruiting phase-2\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Vosoritide<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(111-212 [Canopy HCH-2I])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Hypochondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 2<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to &lt; 36 Months<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with Hypochondroplasia aged 0 to &lt; 36 months over a 52-week period.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measures*<\/h4>\n<ul>\n<li>Incidence of treatment-emergent adverse events and serious adverse events<\/li>\n<li>Change from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry), heart rate, respiratory rate, temperature, blood pressure, and height Z-score<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>0 to &lt; 36 months of age<\/li>\n<li>Weight at Day 1 visit (pre-treatment) &#x2265;3 kg<\/li>\n<li>Have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH)<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Change in height<\/li>\n<li>Cumulative annualized growth velocity (AGV)<\/li>\n<li>6-month interval AGV<\/li>\n<li>Change from baseline in upper to lower body segment ratio<\/li>\n<li>Change from baseline in arm span<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Short stature condition other than HCH<\/li>\n<li>Have an unstable medical condition likely to require surgical intervention during the study period<\/li>\n<li>Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term treatment (&gt; 3 months) at any time<\/li>\n<li>Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<br>\nHCH, Hypochondroplasia;<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT07126262\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT07126262<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-hypochondroplasia filter-tech-observational filter-rec-stat-recruiting observational\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Hypochondroplasia<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(111-902 [Canopy HCH-OS])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Hypochondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Observational<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>A Multicenter Multinational Observational Study of Children With Hypochondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This study will collect growth measurements and other variables of interest to assess growth and the clinical course of hypochondroplasia over time.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measures*<\/h4>\n<ul>\n<li>\nChange in growth (annualized growth velocity, height Z-score, height)\n<\/li>\n<li>\nChange in anthropometric measures (body mass index [BMI], upper and lower length ratio, arms spam to standing height ratio)\n<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>\nPatients &#x2264; 15 years of age\n<\/li>\n<li>\nGenetic confirmation of hypochondroplasia\n<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>\nChange in quality of life and patient and caregiver assessments of disease severity\n<\/li>\n<li>\nRates of medical events of interest\n<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>\nDiagnosis of genetic short stature condition other than hypochondroplasia or a genetic variant known to cause another genetic syndrome associated with short stature\n<\/li>\n<li>\nReceived an investigational product or medical device within the last 6 months\n<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT06212947\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06212947<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-hypochondroplasia filter-tech-peptide filter-rec-stat-enrolling-by-invitation phase-3\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Vosoritide<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(111-308 [Canopy HCH-3 EXT])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Enrolling by Invitation<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Hypochondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 3<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia (CANOPY-HCH-EXT)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Change from baseline in height Z-score<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Completed the Week 52 visit for Study 111-303 or 111-212 and have open epiphyses as assessed by left hand antero-posterior (AP) X-rays<\/li>\n<li>Females &#x2265; 10 years old or who have begun menses must have a negative pregnancy test at the Baseline visit and be willing to have additional pregnancy tests during the study<\/li>\n<li>If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Change from baseline in height<\/li>\n<li>Absolute 6- or 12-month interval AGV<\/li>\n<li>Change from baseline in upper to lower body segment ratio<\/li>\n<li>Change from baseline in Quality of Life In Short-Statured Youth (QoLISSY) total score<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Permanently discontinued study treatment in Study 111-303 or 111-212<\/li>\n<li>Evidence of decreased growth velocity (AGV &lt;1.5 cm\/year) as assessed over a period of at least 6 months and growth plate closure assessed as per standard of care<\/li>\n<li>Planned or expected to have limb-lengthening surgery during the study period<\/li>\n<li>Planned or expected bone-related surgery during the study period<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT07073014?lead=BioMarin%20Pharmaceutical&amp;aggFilters=status:enr%20act%20rec%20unk&amp;rank=1\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT07073014<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-achondroplasia filter-tech-peptide filter-rec-stat-active-not-recruiting phase-2\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Vosoritide<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(111-205 [Canopy ACH-2 EXT])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Active, Not Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Achondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 2<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>A Study to Evaluate Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia (ACH)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This Phase 2, open-label, extension study evaluates the long-term safety, tolerability, and efficacy of BMN 111 (vosoritide) in children with achondroplasia.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Incidence of treatment-emergent adverse events<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Have completed 24 months of vosoritide treatment in Study 111-202<\/li>\n<li>Parent\/guardian provides informed written consent if required<\/li>\n<li>If sexually active, must be willing to use a highly effective method of contraception while participating in the study<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Annualized growth velocity<\/li>\n<li>Height standard score<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Requires any investigational agent prior to completion of study period<\/li>\n<li>Have a condition or circumstance that places the subject at high risk during this study<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT02724228\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT02724228<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-achondroplasia filter-tech-peptide filter-rec-stat-active-not-recruiting phase-2\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Vosoritide<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(111-208 [Canopy ACH-2I EXT])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Active, Not Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Achondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 2<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This Phase 2, open-label, multicenter, long-term extension study, with approximately 70 subjects, evaluates the safety and efficacy of BMN 111 (vosoritide) in children with achondroplasia until subjects reach near-adult final height.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measures*<\/h4>\n<ul>\n<li>Evaluate the incidence of treatment-emergent adverse events<\/li>\n<li>Evaluate change in height\/length z-score in children with ACH treated with vosoritide<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Must have completed Study 111-206 on investigational treatment<\/li>\n<li>Parent\/guardian provides informed written consent if required<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Evaluate the change from baseline in mean annualized growth velocity<\/li>\n<li>Characterize maximum concentration<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Permanently discontinued vosoritide or placebo prior to completion of Study 111-206<\/li>\n<li>Have a clinically significant finding or arrhythmia on ECG that indicates abnormal cardiac function or conduction or QTc-F &gt;450 msec<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<br>\nACH, achondroplasia; ECG, electrocardiogram; QTcF, corrected QT interval by Fridericia.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT03989947\" target=\"_blank\"> For reference and comprehensive trial information, visit ClinicalTrials.gov NCT03989947<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-achondroplasia filter-tech-peptide filter-rec-stat-active-not-recruiting phase-2\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Vosoritide<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(111-209 [Canopy ACH-2H])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Active, Not Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Achondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 2<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This is a Phase 2 randomized, open-label clinical trial of BMN 111 (vosoritide) in infants and young children with a diagnosis of achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Incidence of treatment-emergent adverse events<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Parent\/guardian provides informed written consent if required<\/li>\n<li>Have ACH, documented by genetic testing<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measure*<\/h4>\n<ul>\n<li>Evaluate the effect of vosoritide on total foramen magnum volume<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Have hypochondroplasia or short-stature condition other than achondroplasia<\/li>\n<li>Have CMC that either does not require surgical intervention or does require immediate surgical intervention<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<br>\nACH, achondroplasia; CMC, cervicomedullary compression.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT04554940\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT04554940<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-achondroplasia filter-tech-peptide filter-rec-stat-active-not-recruiting phase-3\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Vosoritide<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(111-302 [Canopy ACH-3 EXT])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Active, Not Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Achondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 3<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This Phase 3, open-label, long-term extension study is intended and designed to assess BMN 111 (vosoritide) as a therapeutic option for the treatment of children with achondroplasia.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Change from baseline in mean annualized growth velocity<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Must have completed Study 111-301<\/li>\n<li>Females &#x2265;10 years old, or those with menses, need negative pregnancy tests at baseline and during the study<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Changes in health-related quality of life<\/li>\n<li>Potential changes in daily activity performance<\/li>\n<li>Characterize maximum concentration of vosoritide in plasma<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Permanently discontinued vosoritide or placebo prior to completion of Study 111-301<\/li>\n<li>Have a clinically significant finding that indicates abnormal cardiac function<\/li>\n<li>Evidence of slowed growth of at least 6 months or closed growth plates in lower extremity X-rays<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/classic.clinicaltrials.gov\/ct2\/show\/NCT03424018\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov  NCT03424018 <\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-hypochondroplasia filter-tech-peptide filter-rec-stat-active-not-recruiting phase-3\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Vosoritide<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(111-303 [Canopy HCH-3])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Active, Not Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Hypochondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 3<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Change from baseline in annualized growth velocity (AGV)<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Patients &ge; 3 to &lt; 18 years of age<\/li>\n<li>Confirmed genetic diagnosis of HCH<\/li>\n<li>A height Z score of &#x2264;-2 standard deviations in reference to the general population of the same age and sex, as calculated using the Center for Disease Control and Prevention (CDC) growth charts<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measure*<\/h4>\n<ul>\n<li>Change from baseline in standing height and height Z-score<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Have an unstable condition likely to require surgical intervention during the study<\/li>\n<li>Evidence of decreased growth velocity and\/or growth plate closure<\/li>\n<li>Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids<\/li>\n<li>Planned or expected to have limb-lengthening surgery or bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction) during the study period<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<br>\nHCH, hypochondroplasia.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT06455059\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06455059<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-hypochondroplasia filter-tech-observational filter-rec-stat-not-started observational\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Hypochondroplasia<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(111-809)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Not started<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Hypochondroplasia<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Observational<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Generating Growth Curves for Standing and Sitting Height in Hypochondroplasia: A Multi-Center Retrospective Natural History Study<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This study will be a retrospective chart review of height measurements in patients &#x2264;18 years old with a clinically or genetically confirmed diagnosis of hypochondroplasia (HCH)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>To develop sex- and age-specific growth curves for height (standing and sitting, as well as lower-to-upper body segment) for patients with HCH<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measure*<\/h4>\n<ul>\n<li>To calculate sex- and age-specific Z-scores (referenced to Centers for Disease Control and Prevention [CDC] average stature) for height (standing and sitting) in patients with HCH<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<sup>12<\/sup><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t                    \n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-listing pipeline-enzyme-therapies\">\n\t\t\t\t\t\t<h2 class=\"h3\">Enzyme Therapies<\/h2>\n\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5 trial-phase trial-phase\">Trial phase<\/h3>\n\n\t\t\t\t\t\t\t<div class=\"pipeline-group\" data-trial-phase-class=\"trial-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">Program (Trial)<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">Condition<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">Phase<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-phenylketonuria filter-tech-enzyme filter-rec-stat-recruiting phase-4\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Pegvaliase<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(165-402 [PALLADIUM])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Phenylketonuria<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 4<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq (PALLADIUM)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This is a Phase 4 study to evaluate the impact of rapid drug desensitization (RDD) on adult (&#x2265;18 years old) participants with PKU who have experienced hypersensitivity reactions (HSRs) leading to treatment interruption or reduction of dose or dosing frequency while receiving commercial Palynziq.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Tolerance, defined as the ability to restart Palynziq at the dose at which the reactive HSR occurred (reactive dose) and escalate as appropriate within 24 weeks following the RDD, without requiring treatment interruption or reduction of dose or dosing frequency due to HSRs; and 2. Reduction in occurrence of HSRs Grade 2 or above within 24 weeks following RDD<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>18 years of age or older<\/li>\n<li>Have phenylketonuria and been receiving commercial Palynziq and enrolled in the REMS<\/li>\n<li>Have developed HSRs leading to treatment interruption or reduction of dose or dosing frequency (Grade 2 or above) while on Palynziq, and be able to undergo RDD within 6 weeks from the reactive HSR and last Palynziq dose<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measure*<\/h4>\n<ul>\n<li>Change in PEG IgG and IgM, PAL IgG and PAL IgM, and C3\/C4 from Baseline to after RDD and at Week 24<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Medical Conditions:<br>\nPregnant on the day of the RDD<br>\nUncontrolled asthma, active upper respiratory infection or other active infections, or cardiovascular disease<\/li>\n<li>Prior\/Concomitant Therapies:<br>\nNot using antihistamine premedication(s) at the time of reactive HSRs<br>\nWilling and able to resume and tolerate Palynziq at the reactive dose prior to the RDD<br>\nReceiving concurrent injectables containing PEG with the exception of PEG-containing vaccines, such as COVID-19 vaccinations<br>\nReceiving beta blockers<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<\/p>\n<p>REMS, Risk Evaluation &amp; Mitigation Strategy; HSR, Hypersensitivity Reactions; PEG, Polyethylene glycol; PAL, Phenylalanine ammonia lyase; IgG, Immunoglobulin G; IgM, Immunoglobulin M;<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT06780332?term=Palladium&amp;rank=2\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06780332 <\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-phenylketonuria filter-tech-enzyme filter-rec-stat-recruiting phase-4\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Pegvaliase<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(165-501[PALace])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Phenylketonuria<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 4<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This is a 10-year multicenter, global, observational study to further characterize the safety profile of pegvaliase, including hypersensitivity reactions, long-term safety and tolerability, and the effectiveness of the additional risk minimization measures (aRMMs) (European Union [EU] only) in subjects receiving pegvaliase for the treatment of PKU.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>To quantify and characterize the risk of protocol-defined safety events in incident-users receiving pegvaliase for the treatment of PKU in a real-world setting<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Documented diagnosis of PKU per local standard of care<\/li>\n<li>Receiving or plan to receive pegvaliase treatment within 30 days of enrollment<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measure*<\/h4>\n<ul>\n<li>To quantify and characterize the risk of protocol-defined safety events in subjects receiving pegvaliase for the treatment of PKU in a real-world setting<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Subject has any kind of disorder that may compromise the ability of the subject to give written informed consent<\/li>\n<li>Currently participating in an interventional study of any investigational product, device, or procedure<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<br>\nPKU, phenylketonuria.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT05813678\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT05813678<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-phenylketonuria filter-tech-observational filter-rec-stat-recruiting observational\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Pegvaliase<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(165-503 [PALisade])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Phenylketonuria<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Observational<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This is a 10-year multi-center, prospective, longitudinal, single arm study evaluating immunologic, inflammatory and laboratory parameters associated with long-term Palynziq treatment in subjects with phenylketonuria (PKU) in the United States (US).<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Immunologic and inflammatory responses associated with occurrences of: acute systemic hypersensitivity reaction, anaphylaxis, angioedema, serum sickness, severe hypersensitivity reaction, severe or persistent arthralgia, severe injection site reaction<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Enrolled at US sites participating in the 165-501 (PALace) study.<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Immunologic and inflammatory responses (immunologic testing, inflammatory markers) associated with occurrences of end-organ function (e.g., kidney, liver) related, immune-mediated adverse drug reactions<\/li>\n<li>The potential association between immunologic responses and blood Phe levels<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Legal incapacity or limited legal capacity without legal guardian representation<\/li>\n<li>Subject is unable or unwilling to provide informed consent for the additional interventional burden of the study (blood sampling)<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT06305234\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06305234<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-phenylketonuria filter-tech-enzyme filter-rec-stat-recruiting phase-4\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Pegvaliase<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p> (165-504 [PALomino])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Phenylketonuria<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 4<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq&#xAE; (Pegvaliase) During Pregnancy and Breastfeeding (PALomino)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This is a Phase 4 observational study designed to assess the impact of Palynziq&#xAE; (pegvaliase) treatment in pregnant women with PKU and on their offspring who were exposed to pegvaliase at any time during pregnancy and breastfeeding.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Pregnancy outcome and infant development for women who have been treated with Palynziq (pegvaliase) within 2 weeks of their last menstrual period<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Subject or legal guardian consent<\/li>\n<li>Confirmation of ongoing pregnancy<\/li>\n<li>Diagnosed with PKU<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Pregnancy outcomes<\/li>\n<li>Serious adverse events<\/li>\n<li>Maternal pegvaliase use during breastfeeding<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Currently participating in a BioMarin-sponsored interventional study<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<br>\nPKU, phenylketonuria.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT05579548\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT05579548<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-cln2-disease filter-tech-observational filter-rec-stat-recruiting observational\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Cerliponase alfa<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(190-504)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>CLN2 Disease<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Observational<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Cerliponase Alfa Observational Study<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>To evaluate long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease).<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<br>\nCLN2, late infantile neuronal ceroid lipofuscinosis type 2;<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/catalogues.ema.europa.eu\/node\/1605\/administrative-details\" target=\"_blank\">For reference and comprehensive trial information, visit EMA.Europa.eu EUPAS29031<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-phenylketonuria filter-tech-enzyme filter-rec-stat-active-not-recruiting phase-3\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Pegvaliase<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(165-306 [PEGASUS])<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Active, Not Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Phenylketonuria<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 3<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria (PEGASUS)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This is a Phase 3, open-label, randomized, controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measures*<\/h4>\n<ul>\n<li>Change in blood Phe concentration<\/li>\n<li>Incidence of treatment-emergent adverse events as assessed by CTCAE v5.0<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Must be 12 to 17 years old<\/li>\n<li>Signed informed consent<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measure*<\/h4>\n<ul>\n<li>Change in total dietary protein intake<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Previous treatment with pegvaliase<\/li>\n<li>Use of any medication that is intended to treat PKU within 14 days prior to administration<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<br>\nCTCAE v5.0, Common Terminology Criteria for Adverse Events version 5.0; Phe, phenylalanine; PKU, phenylketonuria.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT05270837\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT05270837<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-cln2-disease filter-tech-observational filter-rec-stat-active-not-recruiting observational\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Cerliponase alfa<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(190-501)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Active, Not Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>CLN2 Disease<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Observational<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Cerliponase Alfa Observational Study in the US<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This is a multicenter, observational study for patients with a confirmed diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as TPP1 deficiency, who intend to be or are currently being treated with cerliponase alfa.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>To evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis type 2<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Diagnosed with CLN2 disease<\/li>\n<li>Currently receiving or plan to begin treatment with cerliponase alfa<\/li>\n<li>Parent\/guardian provides informed written consent if required<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Hypersensitivity<\/li>\n<li>Impact of severe adverse events on motor and language functions<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Currently receiving treatment in another investigational device or drug study<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<br>\nCLN2, late infantile neuronal ceroid lipofuscinosis type 2; TPP1, tripeptidyl peptidase 1<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT04476862\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT04476862<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t                    \n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-listing pipeline-innovation\">\n\t\t\t\t\t\t<h2 class=\"h3\">Innovation<\/h2>\n\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5 trial-phase trial-phase\">Trial phase<\/h3>\n\n\t\t\t\t\t\t\t<div class=\"pipeline-group\" data-trial-phase-class=\"trial-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">Program (Trial)<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">Condition<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">Phase<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-duchenne-muscular-dystrophy filter-tech-antisense-oligonucleotide filter-rec-stat-recruiting phase-2\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">BMN 351<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(351-201)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Duchenne Muscular Dystrophy<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Phase 2<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>A Phase 1\/&#x200B;2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This Phase 1\/2 clinical study will evaluate the safety and tolerability of BMN 351 in patients with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Incidence of adverse and serious adverse events with BMN 351<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Ambulatory patients 4-10 years of age<\/li>\n<li>Diagnosis of DMD with a mutation amenable to exon 51 skipping<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measure*<\/h4>\n<ul>\n<li>Pharmacokinetic (PK) concentrations of BMN 351 in plasma, urine and muscle<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>History of gene therapy treatment<\/li>\n<li>Use of exon skipping therapy within 12 weeks prior to first visit<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<br>\nDMD, duchenne muscular dystrophy.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT06280209\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06280209<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":2,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"class_list":["post-98","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is 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