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Clinical potential of ataluren in the treatment of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is an autosomal dominant, X-linked neuromuscular disorder caused by mutations in dystrophin, one of the largest genes known to date. Dystrophin gene mutations are generally transmitted from the mother to male offspring and can occur throughout the coding length of t...

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Bibliographic Details
Published in:Degener Neurol Neuromuscul Dis
Main Authors: Namgoong, John Hyun, Bertoni, Carmen
Format: Artigo
Language:Inglês
Published: Dove Medical Press 2016
Subjects:
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6053089/
https://ncbi.nlm.nih.gov/pubmed/30050367
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/DNND.S71808
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