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Article

Dystrophin Analysis in Clinical Trials

1 Jan 2014

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disease with unmet medical need. The disease is caused by mutations that disrupt the open reading frame of the dystrophin protein that is required to maintain muscle fiber stability during contraction. Lacking dystrophin patients' muscle fibers are continuously damaged eventually leading to replacement of muscle tissue by fibrotic and adipose tissues...

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PMID: 27858668
DOI: 10.3233/jnd-140013

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