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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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Bibliographische Detailangaben
Veröffentlicht in:J Neurol Neurosurg Psychiatry
Hauptverfasser: Ricotti, Valeria, Ridout, Deborah A, Pane, Marika, Main, Marion, Mayhew, Anna, Mercuri, Eugenio, Manzur, Adnan Y, Muntoni, Francesco
Format: Artigo
Sprache:Inglês
Veröffentlicht: BMJ Publishing Group 2016
Schlagworte:
Online Zugang: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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