Duchenne muscular dystrophy: A review of corticosteroid-based treatment

Duchenne muscular dystrophy (DMD) is an inherited X-linked recessive form of muscular dystrophy, which affects 1 in 3.500 men, and is caused by a mutation in the gen coding for the protein dystrophin. Most DMD patients show absence of dystrophin. Until a molecular therapy is available for this condition, corticosteroids temporary increase muscular function. Several studies have demonstrated that both prednisone (0.75 mg/kg) and deflazacort (0.9 mg/kg) increase muscular mass and delay DMD progression. The use of corticosteroids delay the loss of independent ambulation, the rate of muscular degeneration, and it also improves cardiac and respiratory function. Aside from their positive effects on motor function preservation, prednisone and deflazacort are associated with significant side effects. Deflazacort has fewer side effects, but the risk of developing cataracts is higher. The beneficial and side effects of corticosteroids must be carefully monitored.