{"id":6873,"date":"2026-06-04T18:26:55","date_gmt":"2026-06-04T18:26:55","guid":{"rendered":"https:\/\/medical.biomarin.com\/en-us\/?page_id=6873"},"modified":"2026-06-08T20:00:36","modified_gmt":"2026-06-08T20:00:36","slug":"noonan-syndrome","status":"publish","type":"page","link":"https:\/\/medical.biomarin.com\/en-us\/noonan-syndrome\/","title":{"rendered":"Noonan Syndrome"},"content":{"rendered":"<div id=\"acf-block-68de3cac2ef74\" class=\"hero hero-no-overlay hero-large-first-para\">\n            <div class=\"hero-background-image hero-background-image-desktop\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/10\/Program-banner-bones.png?v=1.22);\"><\/div>\n\t    <div class=\"hero-background-image hero-background-image-mobile\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/10\/Program-banner-bones-Mobile.png?v=1.22);\"><\/div>\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>Noonan Syndrome\n<\/h1>\n\t\t\t\t\t\t\t\t\t\t\t\t    <p>Learn more about Noonan Syndrome and BioMarin research<\/p>\n\t\t\t\t\t\t\t\t\n                <p class=\"inline-buttons\">\n                                                        <\/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-68e7d6279a7e3\" class=\"block cards cards-2-col\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t\t<div class=\"cards-title\">\n\t\t\t    \t\t\t\t\t\t\t<\/div>\n\t\t\t<div class=\"cards-wrapper\">\n\t\t\t    \n<div id=\"acf-block-68e7d6279ad98\" class=\"card card-product card-fixed-button\" data-muted-autoplay=\"false\">\n        <div class=\"card-content\">\n                            <h4>Clinical Program\n<\/h4>\n        \n                                                                                                                                                                                                                                                                                    \n                                                                                        <\/div>\n                                <div class=\"card-actions\">\n                            \n                            <p><a class=\"button button-arrow\" href=\"#acf-block-68ee5c07eba7b\" target=\"_self\">See clinical trials<\/a><\/p>\n                                                                                    <\/div>\n<\/div>\n\n<div id=\"acf-block-68e7d6279ae37\" class=\"card card-product card-fixed-button\" data-muted-autoplay=\"false\">\n        <div class=\"card-content\">\n                            <h4>Congress Posters and Presentations\n<\/h4>\n        \n                                                                                                                                                                                                                                                                                    \n                                                                                        <\/div>\n                                <div class=\"card-actions\">\n                            \n                            <p><a class=\"button button-arrow\" href=\"#acf-block-68ecfd62c135f\" target=\"_self\">See posters &amp; presentations<\/a><\/p>\n                                                                                    <\/div>\n<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-68ee5c07eba7b\" class=\"vblock pipeline pipeline-listing-wrapper no-pipeline-filters\">\n    <!-- Pipeline Filter Header\n    Integration notes:\n    - Toggle button-filter-active class to filter button when active\n    - Component here is generic filter header and is also used for resources (filters themselves differ)\n    -->\n    <div class=\"block filter-header\">\n        <div class=\"wrapper\">\n            <div class=\"inner-wrapper\">\n                <h1>Noonan Syndrome Pipeline<\/h1>\n                \n                            <\/div>\n        <\/div>\n    <\/div>\n\n    <!-- Research Pipeline block -->\n    <div class=\"block bg-band pipeline-listing-main\">\n        <div class=\"wrapper\">\n            <div class=\"inner-wrapper\">\n                                                                            \n                                        <div class=\"pipeline-listing pipeline-skeletal-conditions\">\n                        <h2 class=\"h3\">Skeletal Conditions<\/h2>\n\n                                                                                                                                        <h3 class=\"h5 trial-phase trial-phase\">Trial phase<\/h3>\n\n                            <div class=\"pipeline-group\" data-trial-phase-class=\"trial-phase\">\n                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                                                            <div class=\"pipeline-headers\">\n                                            <div class=\"pipeline-headers-summary\">\n                                                <div class=\"pipeline-product\">Program (Trial)<\/div>\n                                                <div class=\"pipeline-condition\">Condition<\/div>\n                                                <div class=\"pipeline-technology\">Phase<\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">Pre-IND<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">Phase 1<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">Phase 2<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">Phase 3<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">Phase 4<\/div>\n                                                <div class=\"pipeline-expand\"><\/div>\n                                            <\/div>\n                                        <\/div>\n                                    \n                                    \n                                    <article class=\"pipeline-trial pre-ind phase-1 phase-2 phase-3 filter-condition-noonan-syndrome filter-tech-peptide filter-rec-stat-recruiting\">\n                                        <div class=\"pipeline-summary\">\n                                            <div class=\"pipeline-headers-summary\">\n                                                <div class=\"pipeline-product\">\n                                                    <h3 class=\"h5\">Vosoritide<\/h3>\n                                                    <p>(111-211 [Canopy NS])<\/p>\n                                                    <p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n                                                <\/div>\n                                                                                                    <div class=\"pipeline-condition\">\n                                                        <h4 class=\"h5\">Condition<\/h4>\n                                                        <p>Noonan Syndrome<\/p>\n                                                    <\/div>\n                                                                                                                                                    <div class=\"pipeline-technology\">\n                                                        <h4 class=\"h5\">Phase<\/h4>\n                                                        <ul>\n                                                            <li>Peptide<\/li>\n                                                        <\/ul>\n                                                    <\/div>\n                                                                                                <div class=\"pipeline-details\">\n                                                                                                            <h4>Title<\/h4>\n                                                        <p>A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment<\/p>\n                                                    \n                                                                                                            <h4>Study Description<\/h4>\n                                                        <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                                                    \n                                                                                                            <div class=\"detail-boxes\">\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Change from baseline in annualized growth velocity (AGV)<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <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                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <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                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <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                                                                <\/div>\n                                                                                                                    <\/div>\n                                                    \n                                                    <p>* Additional measures\/criteria may apply.<\/p>\n                                                                                                            <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                                                                                                    <\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases pipeline-inline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">Pre-IND<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">Phase 1<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">Phase 2<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">Phase 3<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">Phase 4<\/div>\n                                                <div class=\"pipeline-expand\"><\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-expand\">\n                                                    <div class=\"trigger\"><\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/article>\n                                                            <\/div>\n                                            <\/div>\n                            <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68ecfd62c135f\" class=\"block resources-listing\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\n                            <div class=\"listing-title\">\n                                            <h2>Congress Posters and Presentations\n<\/h2>\n                                                        <\/div>\n\t\t\t\t\t\t                                    \n                    \n                                                                                                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-poster\">\n                                                    <p class=\"resource-type\">Poster<\/p>\n                                                <div class=\"resource-split-layout\">\n                            <div class=\"resource-image-block\">\n                                <div class=\"resource-image\">\n                                                                            <div class=\"resource-feature-image\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/05\/Congresses-1-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                                                        <p><a class=\"button modal-pdfjs permitted\" href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/09\/ASBMR-2025_Inadequate_Response-poster_MED-BM-0013_SEPT_2025.pdf?v=1.22\" target=\"_blank\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Congresses<\/li>\n                                                                                                    <li>Noonan Syndrome<\/li>\n                                                                                                    <li>ASBMR<\/li>\n                                                                                                    <li>2025<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Criteria for defining inadequate response to growth hormone in Noonan syndrome, Turner syndrome, and SHOX deficiency\n<\/h2>\n                                                                                                <div class=\"resource-links\">\n                                                                                                                                                            \t                                                                                                                                                                        <p><a class=\"button modal-pdfjs permitted\" href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/09\/ASBMR-2025_Inadequate_Response-poster_MED-BM-0013_SEPT_2025.pdf?v=1.22\" target=\"_blank\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-poster\">\n                                                    <p class=\"resource-type\">Poster<\/p>\n                                                <div class=\"resource-split-layout\">\n                            <div class=\"resource-image-block\">\n                                <div class=\"resource-image\">\n                                                                            <div class=\"resource-feature-image\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/05\/Congresses-1-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                                                        <p><a class=\"button modal-pdfjs permitted\" href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/07\/ESPE-ESE_2025-211_NTS_Study_Design-poster-MMRC-ISS-00103_2025_MAY.pdf?v=1.22\" target=\"_blank\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Congresses<\/li>\n                                                                                                    <li>Noonan Syndrome<\/li>\n                                                                                                    <li>ESE<\/li>\n                                                                                                    <li>ESPE<\/li>\n                                                                                                    <li>2025<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Design of a randomized, multicentre, phase 2 study of vosoritide in children with Turner syndrome, Noonan syndrome, or short stature homeobox-containing gene (SHOX) deficiency\n<\/h2>\n                                                                                                <div class=\"resource-links\">\n                                                                                                                                                            \t                                                                                                                                                                        <p><a class=\"button modal-pdfjs permitted\" href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/07\/ESPE-ESE_2025-211_NTS_Study_Design-poster-MMRC-ISS-00103_2025_MAY.pdf?v=1.22\" target=\"_blank\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                            \t\t<\/div>\n\t<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":3,"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-6873","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.7 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ 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