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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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書目詳細資料
發表在:J Neurol Neurosurg Psychiatry
Main Authors: Ricotti, Valeria, Ridout, Deborah A, Pane, Marika, Main, Marion, Mayhew, Anna, Mercuri, Eugenio, Manzur, Adnan Y, Muntoni, Francesco
格式: Artigo
語言:Inglês
出版: BMJ Publishing Group 2016
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在線閱讀: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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