Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is not treatable; there is no cure. More than a decade ago, randomized trials demonstrated that oral steroid therapy was of benefit to DMD patients by prolonging ambulation. Although few significant side effects were reported, study patients were followed for 18 months or less. However, when treating DMD with steroids, the clinician must consider beginning treatment in mid-childhood and continuing until adolescence or longer, a total of at least 10 years. There is no evidence that steroids are associated with prolonged life or with improved pulmonary or cardiac function in DMD. It is clear that the risk of side effects increases with duration of use of oral steroids. Therefore, oral steroids are not recommended for treatment of DMD on a routine basis. If, in certain cases, one does institute therapy, the patient should be monitored carefully for side effects, maintain dietary restriction, and exercise regularly.