Glucocorticoid Corticosteroids for Duchenne Muscular Dystrophy

The review assessed the effects of glucocorticoid corticosteroids on Duchenne muscular dystrophy (DMD), a childhood disease characterized by muscle wasting and loss of walking ability. Evidence from randomized controlled trials with 249 participants indicated that these steroids improved muscle strength and function for 6 months to 2 years, with the most effective prednisolone dose being 0.75 mg/kg/day. Short-term side effects, such as weight gain and behavioral changes, were common but manageable. Long-term benefits and risks remain unclear, though non-randomized studies suggest functional benefits over 5 years. Further randomized trials are needed to evaluate long-term outcomes.