Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial
Rattachement africain : us, gb, it, jp, es, de, ch. Niveau de preuve : code pays fourni par la source.
Le résumé fourni par la source
INTRODUCTION: vector genomes/kg) in ambulatory male patients with DMD aged 4 to < 8 years; N = 125. One-year results demonstrated the manageable safety of delandistrogene moxeparvovec, consistent with previous clinical trials. The primary endpoint (change from baseline in North Star Ambulatory Assessment [NSAA] total score at 52 weeks compared with placebo) did not meet statistical significance. However, key secondary endpoints, comprising timed function tests, suggested slowing or stabilization of disease progression with delandistrogene moxeparvovec, which could become increasingly evident over longer periods of time. We report 2-year follow-up of safety and functional outcomes in patients receiving delandistrogene moxeparvovec in EMBARK part 1. METHODS: As a result of the crossover study design, 2-year functional outcomes of patients receiving delandistrogene moxeparvovec in part 1 of EMBARK were compared, by pre-specified analysis, with a matched propensity score-weighted external control (EC). RESULTS: At 2 years, EMBARK patients showed statistically significant benefit versus the EC cohort in functional outcomes prognostic for delaying loss of ambulation (NSAA, Time to Rise, 10-m Walk/Run), demonstrating sustained stabilization or slowing of disease progression. Delandistrogene moxeparvovec micro-dystrophin expression and sarcolemmal localization were maintained over 64 weeks. No new safety signals were observed between week 52 and week 104. Between baseline and week 104, there were no treatment-related deaths, study discontinuations due to adverse events, or clinically significant complement-mediated adverse events. CONCLUSIONS: At 2 years, stabilization or slowing of DMD disease progression was observed in ambulatory male patients with DMD aged 4 to < 8 years receiving delandistrogene moxeparvovec versus a matched EC cohort. Safety was consistent with EMBARK 1-year data and manageable with appropriate monitoring. CLINICALTRIALS: GOV: NCT05096221.
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Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial
- Date Crossref
- 10/01/2026
- Éditeur
- Springer Science and Business Media LLC
- Type
- journal-article
Ce recoupement confirme des métadonnées liées au DOI. Il ne confirme ni la méthode ni les conclusions de l’étude, et il ne compte pas comme une seconde source scientifique indépendante.
Où se fait cette recherche
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Nationwide Children's Hospital Center for Gene Therapy pays non établi dans la noticeÉtablissement de santé
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Sarepta Therapeutics (United States) pays non établi dans la noticeEntreprise
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Great Ormond Street Hospital pays non établi dans la noticeÉtablissement de santé
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University College London NIHR Great Ormond Street Hospital Biomedical Research Centre pays non établi dans la noticeUniversité ou école supérieure
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UC Davis Health pays non établi dans la noticeÉtablissement de santé
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Centro Clinico Nemo pays non établi dans la noticeÉtablissement de santé
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Istituti di Ricovero e Cura a Carattere Scientifico pays non établi dans la noticeÉtablissement de santé
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University of Rochester Medicine pays non établi dans la noticeÉtablissement de santé
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National Center of Neurology and Psychiatry pays non établi dans la noticeÉtablissement de santé
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University of Florida Department of Pediatrics pays non établi dans la noticeUniversité ou école supérieure
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Hospital Sant Joan de Déu Barcelona Neuropaediatrics Department pays non établi dans la noticeÉtablissement de santé
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Sant Joan de Déu Research Foundation pays non établi dans la noticeStructure de recherche
Center for Gene Therapy — Nationwide Children's Hospital, Sarepta Therapeutics (United States) et Great Ormond Street Hospital, avec 9 autres affiliations.
Une affiliation ne permet pas de déduire la nationalité d’un auteur.