Evolution of Duchenne Muscular Dystrophy Management: From Traditional Therapies to Novel Interventions

Historically, management of Duchenne muscular dystrophy (DMD) relied primarily on corticosteroids, such as prednisone or deflazacort, to slow muscle degeneration. While effective in prolonging ambulation, long-term corticosteroid use is associated with significant challenges, including growth suppression, weight gain, behavioral changes, and bone complications like scoliosis. Multidisciplinary interventions—including physical therapy, occupational therapy, respiratory care, orthopedic support, and cardiac monitoring—play a vital role in maintaining function and quality of life. Persistent unmet needs, including limited long-term efficacy, side effect management, and disease-modifying options, spurred the development of novel FDA-approved therapies targeting the underlying genetic defect in DMD. These advances aim to complement traditional care, address gaps in treatment, and improve patient outcomes across all stages of the disease.