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Placebo‐controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy

OBJECTIVE: This double‐blind, randomized, placebo‐controlled Phase 2 study (NCT01462292) assessed the 24‐week efficacy, safety, tolerability, and pharmacokinetics of two different subcutaneous drisapersen doses, and the 24‐week off‐dose persistent effect, in ambulant Duchenne muscular dystrophy (DMD...

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Detalles Bibliográficos
Autores principales: McDonald, Craig M., Wong, Brenda, Flanigan, Kevin M., Wilson, Rosamund, de Kimpe, Sjef, Lourbakos, Afrodite, Lin, Zhengning, Campion, Giles
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6093847/
https://www.ncbi.nlm.nih.gov/pubmed/30128316
http://dx.doi.org/10.1002/acn3.579