{"id":4698,"date":"2025-07-17T09:41:02","date_gmt":"2025-07-17T09:41:02","guid":{"rendered":"https:\/\/medical.biomarin.com\/en-us\/?page_id=4698"},"modified":"2026-03-04T14:44:29","modified_gmt":"2026-03-04T14:44:29","slug":"duchenne-muscular-dystrophy-dmd","status":"publish","type":"page","link":"https:\/\/medical.biomarin.com\/en-us\/duchenne-muscular-dystrophy-dmd\/","title":{"rendered":"Duchenne muscular dystrophy (DMD)"},"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-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>Duchenne muscular dystrophy (DMD)\n&#xA0;\n<\/h1>\n\t\t\t\t\t\t\t\t\t\t\t\t    <p>Learn more about Duchenne muscular dystrophy (DMD) 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-68e7d627a167c\" target=\"_self\">See 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 congress posters and 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-68e7d627a167c\" 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-\">\n                        <h2 class=\"h3\"><\/h2>\n\n                                                                                                                                        <h3 class=\"h5 trial-phase dmd-clinical-trials\">DMD Clinical Trial(s)<\/h3>\n\n                            <div class=\"pipeline-group\" data-trial-phase-class=\"dmd-clinical-trials\">\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 pre-ind phase-1 phase-2 filter-condition-duchenne-muscular-dystrophy filter-tech-antisense-oligonucleotide 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 351<\/h3>\n                                                    <p>(351-201)<\/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>Duchenne Muscular Dystrophy<\/p>\n                                                    <\/div>\n                                                                                                                                                    <div class=\"pipeline-technology\">\n                                                        <h4 class=\"h5\">Technology<\/h4>\n                                                        <ul>\n                                                            <li>Antisense Oligonucleotide<\/li>\n                                                        <\/ul>\n                                                    <\/div>\n                                                                                                <div class=\"pipeline-details\">\n                                                                                                            <h4>Title<\/h4>\n                                                        <p>A Phase 1\/&#x200B;2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy<\/p>\n                                                    \n                                                                                                            <h4>Study Description<\/h4>\n                                                        <p>This Phase 1\/2 clinical study will evaluate the safety and tolerability of BMN 351 in patients with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.<\/p>\n                                                    \n                                                                                                            <div class=\"detail-boxes\">\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Incidence of adverse and serious adverse events with BMN 351<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Ambulatory patients 4-10 years of age<\/li>\n<li>Diagnosis of DMD with a mutation amenable to exon 51 skipping<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Secondary Outcome Measure*<\/h4>\n<ul>\n<li>Pharmacokinetic (PK) concentrations of BMN 351 in plasma, urine and muscle<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>History of gene therapy treatment<\/li>\n<li>Use of exon skipping therapy within 12 weeks prior to first visit<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                    <\/div>\n                                                    \n                                                    <p>* Additional measures\/criteria may apply.<br>\nDMD, duchenne muscular dystrophy.<\/p>\n                                                                                                            <p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT06280209\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06280209<\/a><\/p>\n                                                                                                    <\/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-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\/2026\/03\/MDA_2026_DMD-QoL_poster_MED-BM-0239_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>Congresses<\/li>\n                                                                                                    <li>Duchenne muscular dystrophy (DMD)<\/li>\n                                                                                                    <li>Muscular Dystrophy Association (MDA) Clinical &amp; Scientific Conference<\/li>\n                                                                                                    <li>2026<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Qualitative interviews with caregivers of children aged 5&#x2013;7 years with Duchenne muscular dystrophy to assess content validity of the proxy&#x2011;completed DMD-QOL\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\/MDA_2026_DMD-QoL_poster_MED-BM-0239_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-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\/2026\/03\/MDA_2026_351-201_Safety-and-Efficacy_poster_MED-BM-0246_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>Congresses<\/li>\n                                                                                                    <li>Duchenne muscular dystrophy (DMD)<\/li>\n                                                                                                    <li>Muscular Dystrophy Association (MDA) Clinical &amp; Scientific Conference<\/li>\n                                                                                                    <li>2026<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Initial results from an open-label phase 1\/2 study of BMN 351, an antisense oligonucleotide for exon 51-skip amenable Duchenne muscular dystrophy\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\/MDA_2026_351-201_Safety-and-Efficacy_poster_MED-BM-0246_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-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\/2026\/03\/MDA_2026_BMN-351_Development_Story_poster_MED-BM-0236_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>Congresses<\/li>\n                                                                                                    <li>Duchenne muscular dystrophy (DMD)<\/li>\n                                                                                                    <li>Muscular Dystrophy Association (MDA) Clinical &amp; Scientific Conference<\/li>\n                                                                                                    <li>2026<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>The development of BMN 351: an exon 51 skipping antisense oligonucleotide for DMD\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\/MDA_2026_BMN-351_Development_Story_poster_MED-BM-0236_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-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\/2026\/03\/MDA_2026_Muscle_Cell_Composition_poster_MED-BM-0240_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>Congresses<\/li>\n                                                                                                    <li>Duchenne muscular dystrophy (DMD)<\/li>\n                                                                                                    <li>Muscular Dystrophy Association (MDA) Clinical &amp; Scientific Conference<\/li>\n                                                                                                    <li>2026<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Transcriptional biomarker for muscle composition measures BMN 351 treatment response in DMD\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\/MDA_2026_Muscle_Cell_Composition_poster_MED-BM-0240_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-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\/WMS_2025_DMD_QoL_poster_MED-ET-0034_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>Duchenne muscular dystrophy (DMD)<\/li>\n                                                                                                    <li>World Muscle Society (WMS)<\/li>\n                                                                                                    <li>2025<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Qualitative interviews with caregivers of children aged 4&#x2013;7 years with Duchenne muscular dystrophy to assess content validity of the proxy-completed DMD-QOL\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\/WMS_2025_DMD_QoL_poster_MED-ET-0034_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\/09\/WMS_2025_Dystrophin_LCMS_poster_MED-ET-0035_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>Duchenne muscular dystrophy (DMD)<\/li>\n                                                                                                    <li>World Muscle Society (WMS)<\/li>\n                                                                                                    <li>2025<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Quantitative measurement of near full-length dystrophin and muscle content normalizer proteins in human muscle by IA&#x2011;UPLC-MS\/MS\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\/WMS_2025_Dystrophin_LCMS_poster_MED-ET-0035_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\/2024\/09\/WMS-2024-exon51-poster_MMRC-DMD-00010.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>Duchenne muscular dystrophy (DMD)<\/li>\n                                                                                                    <li>World Muscle Society (WMS)<\/li>\n                                                                                                    <li>2024<\/li>\n                                                                                                    <li>Preclinical Data<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>A phase 1\/2 clinical trial of the antisense oligonucleotide BMN 351 now actively recruiting boys with exon 51&#x2013;skip-amenable Duchenne muscular dystrophy\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\/2024\/09\/WMS-2024-exon51-poster_MMRC-DMD-00010.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\/2024\/09\/WMS-2024_mRNA-poster_MMRC-DMD-00009.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>Duchenne muscular dystrophy (DMD)<\/li>\n                                                                                                    <li>World Muscle Society (WMS)<\/li>\n                                                                                                    <li>2024<\/li>\n                                                                                                    <li>Preclinical Data<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Development of a next-generation multiplex ddPCR assay for measurement of in-frame dystrophin mRNA in subjects with Duchenne muscular dystrophy treated with BMN 351\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\/2024\/09\/WMS-2024_mRNA-poster_MMRC-DMD-00009.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-4698","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>Duchenne muscular dystrophy (DMD) | BioMarin Medical<\/title>\n<meta name=\"description\" content=\"Learn more about Duchenne muscular dystrophy (DMD) 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\/duchenne-muscular-dystrophy-dmd\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" 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