Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production

Charleston JS, Schnell FJ, Dworzak J, et al. Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. Neurology 2018;90:e2146-e2154 Twelve patients included in the placebo-controlled trial for eteplirsen*1, where followed in an open-label extension study (the patients originally receiving placebo, where as well treated with eteplirsen in the extension study). During evaluation after […]