More Choices For Boys With DMD

Abstract

Lately, staffers at the advocacy group Parent Project Muscular Dystrophy have been fielding a flurry of phone calls from parents asking for help in making a difficult decision for their children. Two similar treatments for a subset of patients with Duchenne muscular dystrophy, a rare and fatal degenerative disease that affects boys, are suddenly much closer to approval than the DMD community had expected. More clinical studies of the drugs—one each from the biotech firms Sarepta Therapeutics and Prosensa Therapeutics—are opening up, and there’s a real possibility that one or both of the treatments could be on the market next year. Deciding which treatment to try—a choice that PPMD, the advocacy group, can’t make—is agonizing. Parents worry that if they pick the wrong drug, the window for their son to benefit from treatment will close. Moreover, scant data exist to guide them. But it’s a choice nonetheless, and one that ...