Pharmacological management of Duchenne muscular dystrophy

Abstract

Duchenne muscular dystrophy is an inherited disorder mainly affecting males causing progressive skeletal and cardiac muscle weakness. The males can additionally have many complications, including cardiomyopathy, respiratory insufficiency, orthopedic, gastrointestinal, and psychological. The mainstay of treatment is corticosteroids that can slow down the progression of symptoms. The long-term side effects of corticosteroid use are a serious concern and tolerability difficult for many. Weight gain, immunosuppression, hirsutism, and behavioral side effects are among them.