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Functional changes in Becker muscular dystrophy: implications for clinical trials in dystrophinopathies

We performed a 1-year longitudinal study of Six Minute Walk Test (6MWT), North Star Ambulatory Assessment (NSAA), and timed function tests in Becker muscular dystrophy (BMD). Skeletal muscle dystrophin was quantified by immunoblot. We grouped deletions ending on exon 45 (“del 45-x”, n = 28) or 51 (“...

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Detalhes bibliográficos
Publicado no:Sci Rep
Main Authors: Bello, Luca, Campadello, Paola, Barp, Andrea, Fanin, Marina, Semplicini, Claudio, Sorarù, Gianni, Caumo, Luca, Calore, Chiara, Angelini, Corrado, Pegoraro, Elena
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5007528/
https://ncbi.nlm.nih.gov/pubmed/27582364
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep32439
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