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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Bibliografiset tiedot
Julkaisussa:J Neurol Neurosurg Psychiatry
Päätekijät: Ricotti, Valeria, Ridout, Deborah A, Pane, Marika, Main, Marion, Mayhew, Anna, Mercuri, Eugenio, Manzur, Adnan Y, Muntoni, Francesco
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: BMJ Publishing Group 2016
Aiheet:
Neuromuscular
Linkit: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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