The Expanding Armamentarium for Treating Boys with Duchenne Muscular Dystrophy

Corticosteroids, the mainstay of pharmacotherapy for Duchenne muscular dystrophy (DMD), delay loss of ambulation and stabilize pulmonary function. Progressive weakness of respiratory muscles nevertheless leads to restrictive lung disease, respiratory complications, and early morbidity and mortality, underscoring the need for additional therapies, especially for boys who cannot tolerate corticosteroid side effects. Investigators now report results of a small, manufacturer-sponsored, phase III clinical trial in which mostly nonambulatory boys, aged 10 to 18 years, were randomized to oral idebenone (300 mg three times daily) or matching placebo for 52 weeks. The primary endpoint was the change in the percent of predicted peak expiratory flow (…