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Natural history of LGMD2A for delineating outcome measures in clinical trials

OBJECTIVE: Limb‐girdle muscular dystophy 2A (LGMD2A, OMIM) is a slowly progressive myopathy caused by the deficiency in calpain 3, a calcium‐dependent cysteine protease of the skeletal muscle. METHODS: In this study, we carried out an observational study of clinical manifestations and disease progre...

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Detalles Bibliográficos
Autores principales: Richard, Isabelle, Hogrel, Jean‐Yves, Stockholm, Daniel, Payan, Christine A. M., Fougerousse, Françoise, Eymard, Bruno, Mignard, Claude, Lopez de Munain, Adolfo, Fardeau, Michel, Urtizberea, Jon Andoni
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2016
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Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4818744/
https://www.ncbi.nlm.nih.gov/pubmed/27081656
http://dx.doi.org/10.1002/acn3.287