Gene Therapy in Duchenne Muscular Dystrophy: Benefits, Limitations, and Clinical Integration

Gene therapy represents a transformative approach in the management of Duchenne muscular dystrophy (DMD), aiming to restore or replace the defective dystrophin gene and address the underlying cause of the disease. Therapies such as delandistrogene moxeparvovec have demonstrated promising data, including increased dystrophin expression and potential stabilization of muscle function, supporting their emerging role in treatment algorithms. While these therapies offer the potential for durable disease modification, limitations include eligibility based on mutation type, immune responses, long-term safety uncertainties, and complex delivery logistics. Families increasingly inquire about gene therapy, reflecting heightened awareness and hope for disease-modifying options. Counseling focuses on balancing potential benefits with risks, clarifying realistic expectations, and emphasizing the need for ongoing monitoring. Integrating gene therapy into clinical practice requires individualized decision-making and collaboration among patients, caregivers, and multidisciplinary care teams to optimize outcomes and long-term management.