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Eteplirsen in the treatment of Duchenne muscular dystrophy
Duchenne muscular dystrophy is a fatal neuromuscular disorder affecting around one in 3,500–5,000 male births that is characterized by progressive muscular deterioration. It is inherited in an X-linked recessive fashion and is caused by loss-of-function mutations in the DMD gene coding for dystrophi...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Dove Medical Press
2017
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5338848/ https://www.ncbi.nlm.nih.gov/pubmed/28280301 http://dx.doi.org/10.2147/DDDT.S97635 |