Duchenne Muscular Dystrophy: An Update, Part I

Although the cloning of the dystrophin gene has led to major advances in the knowledge of the genetic, molecular basis of Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD), and other muscular dystrophies, with mutations of genes encoding the dystrophin-associated glycoprotein complex (DGC), effective therapies are still lacking. This review reports some of the most promising pharmacological, molecular, and cellular approaches to DMD/BMD. Altogether, these exciting developments are just puzzling work hypothesis whose clinical developments are on the way.