Research Progress on the Efficacy and Safety of Deflazacort in the Treatment of Duchenne Muscular Dystrophy

Deflazacort, as a glucocorticoid medication, is conductive to improving motor function and muscle strength, delaying the loss of ambulation, enhancing pulmonary function, reducing the risk of scoliosis, slowing the progression of cardiomyopathy, and increasing survival rates in patients with Duchenne muscular dystrophy (DMD). In February 2017, the U.S. Food and Drug Administration (FDA) approved deflazacort for the treatment of DMD. In May 2024, deflazacort entered Peking Union Medical College Hospital for designated use through the " temporary import" pathway.This article provides an overview of deflazacort from the perspectives of its mechanism of action, pharmacokinetics, clinical efficacy, and adverse effects, aiming to offer a reference for its rational and safe application in clinical practice.