Deflazacort treatment of Duchenne muscular dystrophy

Abstract Objective: We report the long-term effects on muscle strength and side effects with deflazacort in Duchenne muscular dystrophy (DMD). Study design: Boys with DMD between the ages of 7 and 15 years were reviewed retrospectively; 30 had been treated with deflazacort, and 24 had not. Muscle function, pulmonary function, and side effects were compared. Results: The boys not treated with deflazacort stopped walking at 9.8 ± 1.8 years. Seven of 30 treated boys had stopped walking at 12.3 ± 2.7 years ( P P Conclusions: We conclude that deflazacort can preserve gross motor and pulmonary function in boys with DMD with limited side effects. (J Pediatr 2001;138:45-50)