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 drugs have been published to determine the pooled estimates for the effect of exon skipping in treating DMD.

Methods

A systematic review and meta-analysis of double-blind RCTs comparing exon-skipping drugs with placebo in DMD was performed. Trials were identified by searching published and unpublished studies from electronically available databases and clinical trial registries through October 2017. The primary outcomes were changes in the 6-min walk test (6MWT) distance, North Star Ambul

SUBMITTER: Shimizu-Motohashi Y 

PROVIDER: S-EPMC6003096 | biostudies-literature | 2018 Jun

REPOSITORIES: biostudies-literature