Therapy of Genetic Disorders—Novel Therapies for Duchenne Muscular Dystrophy

Seto JT, Bengtsson NE, Chamberlain JS. Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy. Current pediatrics reports 2014;2:102-112.   Abstract Duchenne muscular dystrophy (DMD) is an inherited, progressive muscle wasting disorder caused by mutations in the dystrophin gene. An increasing variety of approaches are moving towards clinical testing that all aim to restore dystrophin […]