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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Pubblicato in:J Neurol Neurosurg Psychiatry
Autori principali: Ricotti, Valeria, Ridout, Deborah A, Pane, Marika, Main, Marion, Mayhew, Anna, Mercuri, Eugenio, Manzur, Adnan Y, Muntoni, Francesco
Natura: Artigo
Lingua:Inglês
Pubblicazione: BMJ Publishing Group 2016
Soggetti:
Neuromuscular
Accesso online: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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