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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...
Autores principales: | Ricotti, Valeria, Ridout, Deborah A, Pane, Marika, Main, Marion, Mayhew, Anna, Mercuri, Eugenio, Manzur, Adnan Y, Muntoni, Francesco |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BMJ Publishing Group
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4752678/ https://www.ncbi.nlm.nih.gov/pubmed/25733532 http://dx.doi.org/10.1136/jnnp-2014-309405 |
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