Abstract
The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.
The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.
Stanley Manne Children's Research Institute
Louis A. Simpson and Kimberly K. Querrey Biomedical Research Center
303 East Superior Street
Chicago, IL 60611
