Addressing Unmet Needs and Evidence Gaps in Duchenne Muscular Dystrophy

Despite recent advances in Duchenne muscular dystrophy (DMD) treatment, significant unmet needs remain. Patients continue to face progressive muscle weakness, respiratory and cardiac complications, and challenges in maintaining independence and quality of life. Caregivers often experience high physical, emotional, and logistical burdens. Clinicians are seeking additional data beyond clinical trials, including long-term safety, real-world effectiveness, patient-reported outcomes, and health economic evaluations, to better guide therapy selection and optimize care. Balancing the cost of newer, disease-modifying therapies with safety and efficacy considerations is a key factor in treatment decision-making, particularly given the high price and variable insurance coverage of emerging interventions. Addressing these gaps requires continued research, comprehensive data collection, and individualized, multidisciplinary care strategies. A focus on patient-centered outcomes, real-world evidence, and sustainable access will be critical for improving long-term management and overall quality of life in DMD.