Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls

BACKGROUND: Duchenne muscular dystrophy (DMD) is a rare, X-linked, fatal, degenerative neuromuscular disease caused by DMD gene mutations. A relationship between exon skipping and dystrophin production in exon 51-amenable patients treated with eteplirsen (EXONDYS 51(®)) is established. Once-weekly e...

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Detalles Bibliográficos
Autores principales: Mendell, Jerry R., Khan, Navid, Sha, Nanshi, Eliopoulos, Helen, McDonald, Craig M., Goemans, Nathalie, Mercuri, Eugenio, Lowes, Linda P., Alfano, Lindsay N.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2021
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8385516/
https://www.ncbi.nlm.nih.gov/pubmed/33523015
http://dx.doi.org/10.3233/JND-200548

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