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

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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Bibliographic Details
Main Authors: Bello, Luca, Campadello, Paola, Barp, Andrea, Fanin, Marina, Semplicini, Claudio, Sorarù, Gianni, Caumo, Luca, Calore, Chiara, Angelini, Corrado, Pegoraro, Elena
Format: Online
Language:English
Published: Nature Publishing Group 2016
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5007528/

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https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5007528/

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