Prednisone Therapy for Duchenne's Muscular Dystrophy

In the past five years, progress in understanding the molecular basis of Duchenne's muscular dystrophy has been substantial. The affected gene in this disease has been cloned, and its protein product, dystrophin, characterized.1 The importance of dystrophin in the pathogenesis of this disorder has been defined: dystrophin is absent from muscle in Duchenne's muscular dystrophy and is usually present but of abnormal size in Becker's muscular dystrophy, a milder variant.2 Unfortunately, these dramatic advances have not yet had an effect on the clinical management of muscular dystrophy. Duchenne's muscular dystrophy remains invariably fatal. The disease is common, occurring in approximately . . .