FDA-Approved Therapies and Personalized Treatment Strategies in Duchenne Muscular Dystrophy

Recent FDA-approved therapies for Duchenne muscular dystrophy (DMD) target the underlying genetic and molecular causes of the disease, offering a shift from traditional symptom-focused management. The rationale for studying these targets stems from the need to slow disease progression, preserve muscle function, and address the unmet needs of patients beyond what corticosteroids can achieve. Selecting the appropriate therapy requires consideration of mutation type, disease stage, patient comorbidities, and anticipated benefit-risk profiles. Therapy sequencing is often individualized, guided by clinical judgment and patient-specific factors. Caregivers play an integral role in decision-making, frequently advocating for treatment options and helping monitor response and tolerability. Collaborative approaches between clinicians, patients, and families are essential to optimize outcomes and align therapy with patient needs and goals.