DMD drug approved in US

Country

United States

A new treatment for Duchenne muscular dystrophy (DMD) has been approved in the US which uses a technology called exon skipping to bypasss a specific genetic mutation in the production of dystrophin, a protein which strengthens muscle fibers and protects them from injury. By skipping the exon, normal amounts of dystrophin protein are produced. DMD is a genetic disorder characterised by dystrophin loss and a progressive deterioration of muscle.