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

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

Abstract 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...

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Bibliographic Details
Main Authors: Yuko Shimizu-Motohashi, Terumi Murakami, En Kimura, Hirofumi Komaki, Norio Watanabe
Format: Article
Language:English
Published: BMC 2018-06-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Eteplirsen
Drisapersen
Randomized controlled trial
Pooled estimates
6-min walk test
Prospectively planned meta-analysis
Online Access:http://link.springer.com/article/10.1186/s13023-018-0834-2

Internet

http://link.springer.com/article/10.1186/s13023-018-0834-2

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