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Gene Therapy for Muscular Dystrophy: Moving the Field Forward

Gene therapy for the muscular dystrophies has evolved as a promising treatment for this progressive group of disorders. While corticosteroids and/or supportive treatments remain standard of care for Duchenne muscular dystrophy (DMD), loss of ambulation, respiratory failure and compromised cardiac fu...

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Detalhes bibliográficos
Publicado no:Pediatr Neurol
Main Authors: Al-Zaidy, Samiah, Rodino-Klapac, Louise, Mendell, Jerry R
Formato: Artigo
Idioma:Inglês
Publicado em: 2014
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4254539/
https://ncbi.nlm.nih.gov/pubmed/25439576
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.pediatrneurol.2014.08.002
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