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Exon skipping for Duchenne muscular dystrophy: a systematic review and meta-analysis

BACKGROUND: Exon skipping has been considered a promising therapeutic approach for Duchenne muscular dystrophy (DMD). Eteplirsen received conditional approval in the United States in 2016. To date, no systematic reviews or meta-analyses of randomized controlled trials (RCTs) of exon skipping drugs h...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Shimizu-Motohashi, Yuko, Murakami, Terumi, Kimura, En, Komaki, Hirofumi, Watanabe, Norio
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2018
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6003096/
https://ncbi.nlm.nih.gov/pubmed/29907124
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-018-0834-2
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