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The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials

OBJECTIVE: With the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is fundamental to understand the natural history of this disorder to properly design clinical trials. The aims of this study were to assess the effects produced on motor function by different DMD genoty...

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Detalles Bibliográficos
Publicado en:J Neurol Neurosurg Psychiatry
Main Authors: Ricotti, Valeria, Ridout, Deborah A, Pane, Marika, Main, Marion, Mayhew, Anna, Mercuri, Eugenio, Manzur, Adnan Y, Muntoni, Francesco
Formato: Artigo
Idioma:Inglês
Publicado: BMJ Publishing Group 2016
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Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4752678/
https://ncbi.nlm.nih.gov/pubmed/25733532
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1136/jnnp-2014-309405
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