Pharmacotherapy of Duchenne Muscular Dystrophy

Abstract

Duchenne muscular dystrophy is a myopathic disease caused by mutations in the dystrophin gene. It is inherited in an X-linked recessive manner from mothers to their sons. The presentation is of progressive muscle weakness in the proximal limb muscles and then the distal muscles of the lower extremities. Eventually the muscle weakness affects the upper extremities. The symptoms begin at around 4 years of age.