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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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Vydáno v:Sci Rep
Hlavní autoři: Bello, Luca, Campadello, Paola, Barp, Andrea, Fanin, Marina, Semplicini, Claudio, Sorarù, Gianni, Caumo, Luca, Calore, Chiara, Angelini, Corrado, Pegoraro, Elena
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2016
Témata:
On-line přístup: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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