Sarepta Therapeutics is seeking to convert the accelerated approval of its therapeutic exon-skippers for Duchenne muscular dystrophy to full despite the drugs’ failure to improve motor function in a confirmatory trial.
Sarepta Therapeutics is seeking to convert the accelerated approval of its therapeutic exon-skippers for Duchenne muscular dystrophy to full despite the drugs’ failure to improve motor function in a confirmatory trial.