Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy

McDonald CM, Wong B, Flanigan KM, et al. Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy. Annals of clinical and translational neurology 2018;5:913-926   Abstract Objective: This double-blind, randomized, placebo-controlled Phase 2 study (NCT01462292) assessed the 24-week efficacy, safety, tolerability, and pharmacokinetics of two different subcutaneous drisapersen doses, and the 24-week off-dose persistent […]