Duchenne muscular dystrophy: pathogenesis and promising therapies.

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a severe, progressive, muscle-wasting disease, characterized by progressive deterioration of skeletal muscle that causes rapid loss of mobility. The failure in respiratory and cardiac muscles is the underlying cause of premature death in most patients with DMD. Mutations in the gene encoding dystrophin result in dystrophin deficiency, which is the underlying pathogenesis of...

Topics

• Humans
• Muscular Dystrophy, Duchenne
• Dystrophin
• Muscle, Skeletal
• Mutation
• Neuromuscular Junction