Duchenne muscular dystrophy: Current treatment and emerging exon skipping and gene therapy approach.

Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder that causes debilitating muscle weakness and atrophy due to a loss of the dystrophin protein. Patients with DMD are commonly diagnosed at about 3-5 years of age and progressively decline until complications of the disease often result in death at about 20 years of age. While there is no current cure for DMD, several treatment options focus on improving the quality of life and slowing progression of symptoms associated with the disease.