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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Detalles Bibliográficos
Autores principales: Ricotti, Valeria, Ridout, Deborah A, Pane, Marika, Main, Marion, Mayhew, Anna, Mercuri, Eugenio, Manzur, Adnan Y, Muntoni, Francesco
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2016
Materias:
Neuromuscular
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

Internet

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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