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Exon-skipping Therapy for Muscular Dystrophy: A Roadblock, Detour, or Bump in the Road?
Duchenne muscular dystrophy (DMD) affects 1in 5,000 newborn males. These boys appear healthy as infants and young children, but then experience a heartbreaking decline, as muscle tissue gradually wastes away, leaving patients nonambulant by their late teens. The heart and respiratory muscles are sim...
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| Publicado no: | Sci Transl Med |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2014
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4464785/ https://ncbi.nlm.nih.gov/pubmed/24695683 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.3008873 |
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