Corticosteroids in Duchenne Muscular Dystrophy—A Deflazacort Review

Abstract

Duchenne Muscular dystrophy (DMD) is a devastating disorder that is the most common and severe form of childhood onset muscular dystrophy. While guidelines exist that promote a multidisciplinary approach to treatment of DMD, surveys have identified inconsistent adherence to the recommendations. This is particularly highlighted by the variability in the prescriptive habits of corticosteroids, which have a long history of literature supporting their use in DMD. Relatively recent publications have uncovered differences between countries in the prescription patterns of steroid regimens. Currently, treatment recommendations lack comparative data on the efficacy and safety of alternative dosing regimens of corticosteroids. New research is being conducted to help understand the benefits and risks of the most commonly used glucocorticoid regimens in DMD. Deflazacort is a glucocorticoid corticosteroid that is currently seeking approval in the U.S. This article summarizes the relevant published literature on deflazacort to help inform DMD-treating physicians on its impact on the disease.