Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial

Abstract Introduction Delandistrogene moxeparvovec is a recombinant adeno-associated virus rhesus isolate serotype 74 vector-based gene therapy that addresses the absence of functional dystrophin in Duchenne muscular dystrophy (DMD). EMBARK is a phase 3, two-part, crossover, randomized, placebo-cont...

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Principais autores:	Jerry R. Mendell, Francesco Muntoni, Craig M. McDonald, Eugenio M. Mercuri, Emma Ciafaloni, Hirofumi Komaki, Carmen Leon-Astudillo, Andrés Nascimento, Crystal Proud, Ulrike Schara-Schmidt, Aravindhan Veerapandiyan, Craig M. Zaidman, Matthew Furgerson, Kai Ding, Preeti Singh, Rachael Potter, Damon R. Asher, Alexander P. Murphy, Carol Reid, Gregory Hooper, Carmen O. Torre, Marianna Manfrini, Louise R. Rodino-Klapac
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Adis, Springer Healthcare 2026-01-01
Colecção:	Neurology and Therapy
Assuntos:	Delandistrogene moxeparvovec DMD Duchenne muscular dystrophy Gene therapy Neuromuscular disorders
Acesso em linha:	https://doi.org/10.1007/s40120-025-00879-8
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