{"id":5683,"date":"2025-10-14T13:14:07","date_gmt":"2025-10-14T13:14:07","guid":{"rendered":"https:\/\/medical.biomarin.com\/en-us\/?page_id=5683"},"modified":"2026-03-10T21:33:54","modified_gmt":"2026-03-10T21:33:54","slug":"enpp1-deficiency","status":"publish","type":"page","link":"https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/","title":{"rendered":"ENPP1 Deficiency"},"content":{"rendered":"<div id=\"acf-block-68ee4c9fc0b88\" 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-bubbles.png?v=1.20);\"><\/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-bubbles-Mobile.png?v=1.20);\"><\/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>ENPP1 Deficiency\n<\/h1>\n\t\t\t\t\t\t\t\t\t\t\t\t    <p>Learn more about ENPP1 Deficiency 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-69b08e40e92cd\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68945a7e61a3d\" class=\"block cards cards-3-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-68945a7e61f51\" class=\"card card-fixed-button\" data-muted-autoplay=\"false\">\n        <div class=\"card-content\">\n                            <h4>Publications\n<\/h4>\n        \n                                                                                                            <p>View and access a broad range of publications<\/p>\n                                                                                                                                                                                                                                                                        \n                                                                                        <\/div>\n                                <div class=\"card-actions\">\n                            \n                            <p><a class=\"button button-arrow\" href=\"https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/enpp1-deficiency-publications\/\" target=\"_self\">Publications<\/a><\/p>\n                                                                                    <\/div>\n<\/div>\n\n<div id=\"acf-block-68945a7e61fd8\" class=\"card card-fixed-button\" data-muted-autoplay=\"false\">\n        <div class=\"card-content\">\n                            <h4>Congress Materials\n<\/h4>\n        \n                                                                                                            <p>Access and download posters and presentations from recent congresses<\/p>\n                                                                                                                                                                                                                                                                        \n                                                                                        <\/div>\n                                <div class=\"card-actions\">\n                            \n                            <p><a class=\"button button-arrow\" href=\"#acf-block-68ee5c07ebab5\" target=\"_self\">Posters &amp; Presentations<\/a><\/p>\n                                                                                    <\/div>\n<\/div>\n\n<div id=\"acf-block-68945a7e6205f\" class=\"card card-fixed-button\" data-muted-autoplay=\"false\">\n        <div class=\"card-content\">\n                            <h4>Educational Materials\n<\/h4>\n        \n                                                                                                            <p>View a range of topical and relevant educational materials<\/p>\n                                                                                                                                                                                                                                                                        \n                                                                                        <\/div>\n                                <div class=\"card-actions\">\n                            \n                            <p><a class=\"button button-arrow\" href=\"#acf-block-69b036aa8b929\" target=\"_self\">Educational Materials<\/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>Pipeline and Clinical Development Programs<\/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-innovation\">\n                        <h2 class=\"h3\">Innovation<\/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\">Product (Trial)<\/div>\n                                                <div class=\"pipeline-condition\">Condition<\/div>\n                                                <div class=\"pipeline-technology\">Technology<\/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 filter-condition-enpp1-deficiency-abcc6-deficiency filter-tech-enzyme filter-rec-stat-recruiting\">\n                                        <div class=\"pipeline-summary\">\n                                            <div class=\"pipeline-headers-summary\">\n                                                <div class=\"pipeline-product\">\n                                                    <h3 class=\"h5\">BMN 401 <br> (formerly INZ-701)<\/h3>\n                                                    <p>(701-007 [PROPEL])<\/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>ENPP1 Deficiency, ABCC6 Deficiency<\/p>\n                                                    <\/div>\n                                                                                                                                                    <div class=\"pipeline-technology\">\n                                                        <h4 class=\"h5\">Technology<\/h4>\n                                                        <ul>\n                                                            <li>Enzyme<\/li>\n                                                        <\/ul>\n                                                    <\/div>\n                                                                                                <div class=\"pipeline-details\">\n                                                                                                            <h4>Title<\/h4>\n                                                        <p>PROPEL &#x2013; A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency<\/p>\n                                                    \n                                                                                                            <h4>Study Description<\/h4>\n                                                        <p>The purpose of this prospective registry is to characterize the natural history of ectonucleotide pyrophosphatase\/phosphodiesterase1(ENPP1) Deficiency and the infantile-onset form of adenosine triphosphate (ATP) binding cassette transporter protein subfamily C member 6 (ABCC6) Deficiency longitudinally.<\/p>\n                                                    \n                                                                                                            <div class=\"detail-boxes\">\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Assessments will be collected during each subject&#x2019;s routine visit. The assessments will be done per local standard of care.\n<ul>\n<li>Assessment of Patient Functional changes through a validated Patient Reported Outcomes (PROs) tools.*<\/li>\n<li>Assessment of Health-Related Quality-of-Life (HRQoL) changes through validated PROs tools.*<\/li>\n<li>Measurement of inorganic phosphate (PPi) levels in patients&#x2019; venous blood.*<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Agree to provide access to relevant medical records<\/li>\n<li>One of the following genetic or clinical criteria:\n<ul>\n<li>A confirmed prenatal or postnatal molecular genetic diagnosis of ENPP1 Deficiency with biallelic mutations (i.e., homozygous or compound heterozygous)<\/li>\n<li>A confirmed monoallelic ENPP1 mutation with clinical symptoms<\/li>\n<li>A confirmed prenatal or postnatal molecular genetic diagnosis of ABCC6 Deficiency with biallelic mutations<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Secondary Outcome Measure*<\/h4>\n<ul>\n<li>Not applicable<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Patients who are currently participating in an BMN 401 (INZ-701) interventional clinical study.<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                    <\/div>\n                                                    \n                                                    <p>* Additional measures\/criteria may apply.; All PROs and PPi sampling are optional.<\/p>\n                                                                                                            <p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT06302439\" target=\"_blank\">For comprehensive trial information, visit ClinicalTrials.gov NCT06302439<\/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-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">\n                                                    <div class=\"phase-progress phase-progress-0\"><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                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                    \n                                    \n                                    <article class=\"pipeline-trial pre-ind phase-1 filter-condition-enpp1-deficiency-abcc6-deficiency filter-tech-enzyme filter-rec-stat-recruiting\">\n                                        <div class=\"pipeline-summary\">\n                                            <div class=\"pipeline-headers-summary\">\n                                                <div class=\"pipeline-product\">\n                                                    <h3 class=\"h5\">BMN 401 <br> (formerly INZ-701)<\/h3>\n                                                    <p>(701-104 [ENERGY])<\/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>ENPP1 Deficiency, ABCC6 Deficiency<\/p>\n                                                    <\/div>\n                                                                                                                                                    <div class=\"pipeline-technology\">\n                                                        <h4 class=\"h5\">Technology<\/h4>\n                                                        <ul>\n                                                            <li>Enzyme<\/li>\n                                                        <\/ul>\n                                                    <\/div>\n                                                                                                <div class=\"pipeline-details\">\n                                                                                                            <h4>Title<\/h4>\n                                                        <p>The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency<\/p>\n                                                    \n                                                                                                            <h4>Study Description<\/h4>\n                                                        <p>The primary purpose of this study is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency<\/p>\n                                                    \n                                                                                                            <div class=\"detail-boxes\">\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Primary Outcome Measures*<\/h4>\n<ul>\n<li>Number of Treatment Emergent Adverse Events<\/li>\n<li>Incidence of Anti-Drug Antibodies<\/li>\n<li>Left Ventricular Ejection Fraction<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous)<\/li>\n<li>Have clinical manifestations of Generalized Arterial Calcification of Infancy (GACI) or GACI-2, which may include, but are not limited to, pathologic ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, and cardiomegaly<\/li>\n<li>&lt;1 year of age at Baseline (Day 1)<\/li>\n<li>Weigh &#x2265;0.5 kg at the time of the first dose of INZ-701<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels<\/li>\n<li>Area under the Plasma Concentration versus Time Curve (AUC)<\/li>\n<li>Maximum Plasma Concentration (Cmax)<\/li>\n<\/ul>\n<p>&#xA0;<\/p>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Care has been withdrawn or study participant is receiving end of life care or hospice only<\/li>\n<li>Known malignancy<\/li>\n<li>Receipt of any non-Inozyme investigational new drug within 5 half-lives of the last dose of the other investigational product or within 4 weeks prior to the first dose of INZ-701, whichever is longer, or use of an investigational device, through completion of participation in the study<\/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\/NCT05734196\" target=\"_blank\">For comprehensive trial information, visit ClinicalTrials.gov NCT05734196<\/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-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">\n                                                    <div class=\"phase-progress phase-progress-0\"><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                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                    \n                                    \n                                    <article class=\"pipeline-trial pre-ind phase-1 phase-2 phase-3 filter-condition-enpp1-deficiency filter-tech-enzyme filter-rec-stat-active-not-recruiting\">\n                                        <div class=\"pipeline-summary\">\n                                            <div class=\"pipeline-headers-summary\">\n                                                <div class=\"pipeline-product\">\n                                                    <h3 class=\"h5\">BMN 401 <br> (formerly INZ-701)<\/h3>\n                                                    <p>(701-106 [ENERGY3])<\/p>\n                                                    <p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Active, Not Recruiting<\/span><\/p>\n                                                <\/div>\n                                                                                                    <div class=\"pipeline-condition\">\n                                                        <h4 class=\"h5\">Condition<\/h4>\n                                                        <p>ENPP1 Deficiency<\/p>\n                                                    <\/div>\n                                                                                                                                                    <div class=\"pipeline-technology\">\n                                                        <h4 class=\"h5\">Technology<\/h4>\n                                                        <ul>\n                                                            <li>Enzyme<\/li>\n                                                        <\/ul>\n                                                    <\/div>\n                                                                                                <div class=\"pipeline-details\">\n                                                                                                            <h4>Title<\/h4>\n                                                        <p>The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency<\/p>\n                                                    \n                                                                                                            <h4>Study Description<\/h4>\n                                                        <p>The primary purpose of this study is to assess the efficacy and safety of INZ-701 in children with ENPP1 Deficiency<\/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 Plasma Inorganic Pyrophosphate (PPi) concentration<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous)<\/li>\n<li>&ge;1 year and &lt;13 years of age at Study Day 1<\/li>\n<li>Open growth plates of the distal femur and proximal tibia in both legs<\/li>\n<li>Plasma Inorganic Pyrophosphate (PPi) concentration of &lt;1400 nM<\/li>\n<li>25-hydroxyvitamin D (25[OH]D) levels of &#x2265;12 ng\/mL<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Change from Baseline in skeletal abnormalities as measured by the Radiographic Global Impression of Change (RGI-C) global score<\/li>\n<li>Change from Baseline in rickets as measured by Rickets Severity Score (RSS) total score<\/li>\n<li>Change from Baseline in growth Z-score (height\/body length and weight)<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Receiving any of the following prohibited medications as indicated in the protocol: systemic corticosteroids (&gt;5 mg prednisone equivalent per day), anti-fibroblast growth factor 23 (FGF23), and oral and\/or IV bisphosphonates<\/li>\n<li>Unable or unwilling to discontinue calcitriol or other active forms of vitamin D3 (or analogs) within 7 days prior to Study Day 1 and\/or oral phosphate supplements within 36 hours prior to Study Day 1 if randomized to the INZ-701 arm<\/li>\n<li>Planned orthopedic surgery that may confound the interpretation of study results during the Randomized Treatment Period<\/li>\n<li>Concurrent participation in another interventional clinical study and\/or has received an investigational drug within 5 half-lives of the last dose or within 4 weeks prior to Randomization, whichever is longer, or use of an investigational device<\/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\/NCT06046820\" target=\"_blank\">For comprehensive trial information, visit ClinicalTrials.gov NCT06046820<\/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                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                    \n                                    \n                                    <article class=\"pipeline-trial pre-ind phase-1 phase-2 filter-condition-enpp1-deficiency-abcc6-deficiency filter-tech-enzyme filter-rec-stat-recruiting\">\n                                        <div class=\"pipeline-summary\">\n                                            <div class=\"pipeline-headers-summary\">\n                                                <div class=\"pipeline-product\">\n                                                    <h3 class=\"h5\">BMN 401 <br> (formerly INZ-701)<\/h3>\n                                                    <p>(701-304 [ADAPT])<\/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>ENPP1 Deficiency, ABCC6 Deficiency<\/p>\n                                                    <\/div>\n                                                                                                                                                    <div class=\"pipeline-technology\">\n                                                        <h4 class=\"h5\">Technology<\/h4>\n                                                        <ul>\n                                                            <li>Enzyme<\/li>\n                                                        <\/ul>\n                                                    <\/div>\n                                                                                                <div class=\"pipeline-details\">\n                                                                                                            <h4>Title<\/h4>\n                                                        <p>ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency<\/p>\n                                                    \n                                                                                                            <h4>Study Description<\/h4>\n                                                        <p>The purpose of this study is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition<\/p>\n                                                    \n                                                                                                            <div class=\"detail-boxes\">\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Primary Outcome Measures*<\/h4>\n<ul>\n<li>Number of Treatment Emergent Adverse Events<\/li>\n<li>Incidence of Anti-Drug Antibodies<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>&gt;1 year of age<\/li>\n<li>Completed the protocol-required safety and pharmacokinetic\/pharmacodynamic and\/or efficacy period(s) of a previous INZ-701 clinical study in ENPP1 or ABCC6 Deficiency<\/li>\n<li>Females of childbearing potential who are sexually active must be using or agree to use 1 highly effective form of contraception (per CTFG 2020) from at least 1 month before the first dose of INZ-701 through 30 days after last dose of INZ-701 (greater than 5 half-lives of INZ-701); participants must agree to not donate ova from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701<\/li>\n<li>Males who are sexually active must agree to use condoms from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701; participants must agree to not donate sperm from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Mean change from Baseline in Plasma Inorganic Pyrophosphate (PPi) concentratio<\/li>\n<li>Time to maximum serum concentration (Tmax)<\/li>\n<li>Maximum Plasma Concentration (Cmax)<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Concurrent participation in another interventional clinical study and\/or has received an investigational drug other than INZ-701 within 5 half-lives or within 4 weeks prior to the first dose of INZ-701 in this study, whichever is longer, or use of an investigational device<\/li>\n<li>Pregnant, trying to become pregnant, or breastfeeding<\/li>\n<li>Males trying to father a child<\/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\/NCT06462547\" target=\"_blank\">For comprehensive trial information, visit ClinicalTrials.gov NCT06462547<\/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-0\"><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-68ee5c07ebab5\" 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\/10\/ASBMR-2025_ENPP1_Rickets_poster_MED-BM-0161_OCT2025.pdf?v=1.20\" 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>ENPP1\/ABCC6 Deficiency<\/li>\n                                                                                                    <li>American Society for Bone &amp; Mineral Research (ASBMR)<\/li>\n                                                                                                    <li>2025<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Quantitative evaluation of hypophosphatemic rickets due to ENPP1 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\/10\/ASBMR-2025_ENPP1_Rickets_poster_MED-BM-0161_OCT2025.pdf?v=1.20\" 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\/10\/ASBMR-2025_Patient_caregiver_survey_poster_MED-BM-0160_OCT2025.pdf?v=1.20\" 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>ENPP1\/ABCC6 Deficiency<\/li>\n                                                                                                    <li>American Society for Bone &amp; Mineral Research (ASBMR)<\/li>\n                                                                                                    <li>2025<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Patient and caregiver insights on the real-world burden of the rare genetic disorders, ENPP1 deficiency and ABCC6 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\/10\/ASBMR-2025_Patient_caregiver_survey_poster_MED-BM-0160_OCT2025.pdf?v=1.20\" 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\/10\/ASBMR-2025_PPi_correlations_poster_MED-BM-0162_OCT2025.pdf?v=1.20\" 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>ENPP1\/ABCC6 Deficiency<\/li>\n                                                                                                    <li>American Society for Bone &amp; Mineral Research (ASBMR)<\/li>\n                                                                                                    <li>2025<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Plasma pyrophosphate (PPi) levels correlate with severity of clinical manifestations of ENPP1 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\/10\/ASBMR-2025_PPi_correlations_poster_MED-BM-0162_OCT2025.pdf?v=1.20\" 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\/11\/ESPN_2025_ENPP1_Infant_poster_March2025_MED-ET-0115.pdf?v=1.20\" 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>ENPP1 Deficiency<\/li>\n                                                                                                    <li>European Society for Pediatric Nephrology (ESPN)<\/li>\n                                                                                                    <li>2025<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Impact of the enzyme replacement therapy, INZ-701, in children with ENPP1 deficiency: Experience from an expanded access program\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\/11\/ESPN_2025_ENPP1_Infant_poster_March2025_MED-ET-0115.pdf?v=1.20\" target=\"_blank\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-presentation\">\n                                                    <p class=\"resource-type\">Presentation<\/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\/11\/ECTS_2024_presentation_MED-ET-0092_Nov2025.pdf?v=1.20\" 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>ENPP1 Deficiency<\/li>\n                                                                                                    <li>European Calcified Tissue Society (ECTS)<\/li>\n                                                                                                    <li>2024<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Impact of INZ-701 on bone and mineral metabolism biomarkers and clinical outcomes in adults with ENPP1 deficiency &#x2013; Results from 48-week phase 1\/2 open label study\n\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\/11\/ECTS_2024_presentation_MED-ET-0092_Nov2025.pdf?v=1.20\" target=\"_blank\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                            \t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-69b036aa8b929\" class=\"block resources-listing\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\n                            <div class=\"listing-title\">\n                                            <h2>Educational Materials\n<\/h2>\n                                                        <\/div>\n\t\t\t\t\t\t                                    \n                    \n                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-article\">\n                                                    <p class=\"resource-type\">Resource<\/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\/2024\/11\/Educational-Materials-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\/2026\/03\/ENPP1_THINK-ARHR2-card_MED-BM-0260_MARCH-2026.pdf?v=1.20\" target=\"_blank\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Educational Materials<\/li>\n                                                                                                    <li>ENPP1 Deficiency<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>ARHR2 Clinical Presentation &amp; Diagnosis\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\/2026\/03\/ENPP1_THINK-ARHR2-card_MED-BM-0260_MARCH-2026.pdf?v=1.20\" target=\"_blank\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-article\">\n                                                    <p class=\"resource-type\">Resource<\/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\/2024\/11\/Educational-Materials-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\/2026\/03\/ENPP1_THINK-GACI-card_MED-ET-0176_MARCH-2026.pdf?v=1.20\" target=\"_blank\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Educational Materials<\/li>\n                                                                                                    <li>ENPP1 Deficiency<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>GACI Clinical Presentation &amp; Diagnosis\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\/2026\/03\/ENPP1_THINK-GACI-card_MED-ET-0176_MARCH-2026.pdf?v=1.20\" target=\"_blank\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-article\">\n                                                    <p class=\"resource-type\">Resource<\/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\/2024\/11\/Educational-Materials-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\/2026\/03\/ENPP1_Ed-Material_ENERGY1_studycard_MED-ET-0177-MARCH_2026.pdf?v=1.20\" target=\"_blank\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Educational Materials<\/li>\n                                                                                                    <li>ENPP1 Deficiency<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>The ENERGY Study Investigating BMN 401*, a subcutaneous ENPP1 enzyme therapy, for infants with GACI\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\/2026\/03\/ENPP1_Ed-Material_ENERGY1_studycard_MED-ET-0177-MARCH_2026.pdf?v=1.20\" 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":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-5683","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.1.1 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>ENPP1 Deficiency | BioMarin Medical<\/title>\n<meta name=\"description\" content=\"Learn more about ENPP1 Deficiency and BioMarin research\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"ENPP1 Deficiency | BioMarin Medical\" \/>\n<meta property=\"og:description\" content=\"Learn more about ENPP1 Deficiency and BioMarin research\" \/>\n<meta property=\"og:url\" content=\"https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/\" \/>\n<meta property=\"og:site_name\" content=\"BioMarin Medical\" \/>\n<meta property=\"article:modified_time\" content=\"2026-03-10T21:33:54+00:00\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/\",\"url\":\"https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/\",\"name\":\"ENPP1 Deficiency | BioMarin Medical\",\"isPartOf\":{\"@id\":\"https:\/\/medical.biomarin.com\/en-us\/#website\"},\"datePublished\":\"2025-10-14T13:14:07+00:00\",\"dateModified\":\"2026-03-10T21:33:54+00:00\",\"description\":\"Learn more about ENPP1 Deficiency and BioMarin research\",\"breadcrumb\":{\"@id\":\"https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/#breadcrumb\"},\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/\"]}]},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https:\/\/medical.biomarin.com\/en-us\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"ENPP1 Deficiency\"}]},{\"@type\":\"WebSite\",\"@id\":\"https:\/\/medical.biomarin.com\/en-us\/#website\",\"url\":\"https:\/\/medical.biomarin.com\/en-us\/\",\"name\":\"BioMarin Medical\",\"description\":\"\",\"potentialAction\":[{\"@type\":\"SearchAction\",\"target\":{\"@type\":\"EntryPoint\",\"urlTemplate\":\"https:\/\/medical.biomarin.com\/en-us\/?s={search_term_string}\"},\"query-input\":{\"@type\":\"PropertyValueSpecification\",\"valueRequired\":true,\"valueName\":\"search_term_string\"}}],\"inLanguage\":\"en-US\"}]}<\/script>\n<!-- \/ Yoast SEO plugin. -->","yoast_head_json":{"title":"ENPP1 Deficiency | BioMarin Medical","description":"Learn more about ENPP1 Deficiency and BioMarin research","robots":{"index":"index","follow":"follow","max-snippet":"max-snippet:-1","max-image-preview":"max-image-preview:large","max-video-preview":"max-video-preview:-1"},"canonical":"https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/","og_locale":"en_US","og_type":"article","og_title":"ENPP1 Deficiency | BioMarin Medical","og_description":"Learn more about ENPP1 Deficiency and BioMarin research","og_url":"https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/","og_site_name":"BioMarin Medical","article_modified_time":"2026-03-10T21:33:54+00:00","twitter_card":"summary_large_image","schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"WebPage","@id":"https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/","url":"https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/","name":"ENPP1 Deficiency | BioMarin Medical","isPartOf":{"@id":"https:\/\/medical.biomarin.com\/en-us\/#website"},"datePublished":"2025-10-14T13:14:07+00:00","dateModified":"2026-03-10T21:33:54+00:00","description":"Learn more about ENPP1 Deficiency and BioMarin research","breadcrumb":{"@id":"https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/#breadcrumb"},"inLanguage":"en-US","potentialAction":[{"@type":"ReadAction","target":["https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/"]}]},{"@type":"BreadcrumbList","@id":"https:\/\/medical.biomarin.com\/en-us\/enpp1-deficiency\/#breadcrumb","itemListElement":[{"@type":"ListItem","position":1,"name":"Home","item":"https:\/\/medical.biomarin.com\/en-us\/"},{"@type":"ListItem","position":2,"name":"ENPP1 Deficiency"}]},{"@type":"WebSite","@id":"https:\/\/medical.biomarin.com\/en-us\/#website","url":"https:\/\/medical.biomarin.com\/en-us\/","name":"BioMarin Medical","description":"","potentialAction":[{"@type":"SearchAction","target":{"@type":"EntryPoint","urlTemplate":"https:\/\/medical.biomarin.com\/en-us\/?s={search_term_string}"},"query-input":{"@type":"PropertyValueSpecification","valueRequired":true,"valueName":"search_term_string"}}],"inLanguage":"en-US"}]}},"_links":{"self":[{"href":"https:\/\/medical.biomarin.com\/en-us\/wp-json\/wp\/v2\/pages\/5683","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/medical.biomarin.com\/en-us\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/medical.biomarin.com\/en-us\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/medical.biomarin.com\/en-us\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/medical.biomarin.com\/en-us\/wp-json\/wp\/v2\/comments?post=5683"}],"version-history":[{"count":65,"href":"https:\/\/medical.biomarin.com\/en-us\/wp-json\/wp\/v2\/pages\/5683\/revisions"}],"predecessor-version":[{"id":6700,"href":"https:\/\/medical.biomarin.com\/en-us\/wp-json\/wp\/v2\/pages\/5683\/revisions\/6700"}],"wp:attachment":[{"href":"https:\/\/medical.biomarin.com\/en-us\/wp-json\/wp\/v2\/media?parent=5683"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}