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

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 o...

Source: Orphanet Journal of Rare Diseases - June 15, 2018 Category: Internal Medicine Authors: Yuko Shimizu-Motohashi, Terumi Murakami, En Kimura, Hirofumi Komaki and Norio Watanabe Tags: Research Source Type: research