Key Clinical Trial Endpoints in Duchenne Muscular Dystrophy

In evaluating clinical trials for Duchenne muscular dystrophy (DMD), selecting meaningful endpoints is critical for assessing therapeutic impact. Functional measures, such as the 6-minute walk test, timed motor milestones, and upper limb assessments, are highly valued because they directly reflect patients’ mobility, independence, and daily functioning. Respiratory and cardiac endpoints are also essential, as progressive pulmonary and cardiac decline are major contributors to morbidity and mortality in DMD. Biomarker-based outcomes, including dystrophin expression levels, serum creatine kinase, and imaging-based muscle composition assessments, provide objective insights into disease modification. Patient-reported outcomes and quality-of-life measures are increasingly recognized for capturing the lived experiences of patients and caregivers. Prioritizing endpoints that balance clinical relevance, sensitivity to change, and patient-centeredness ensures that trial results meaningfully inform treatment decisions, guide regulatory approvals, and ultimately improve long-term outcomes for individuals with DMD.