ABSTRACT
The identification of the genetic basis for Duchenne muscular dystrophy (DMD) in 1987 understandably raised hopes that meaningful therapy for this disorder was just around the corner (1,2). Aside from the excitement generated by the possibility of ‘‘gene therapy,’’ there was cautious optimism that defining the molecular pathogenesis of the disease might lead to pharmacotherapies that could interrupt the inexorable muscle deterioration associated with this disorder. These hopes were raised even higher two years later when a prospective, randomized, placebo-controlled trial demonstrated that prednisone improved strength in DMD (3).
