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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...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Dove Medical Press
2016
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6053089/ https://www.ncbi.nlm.nih.gov/pubmed/30050367 http://dx.doi.org/10.2147/DNND.S71808 |