Considerations in establishing meaningful clinical endpoints in Duchenne muscular dystrophy & other neuromuscular disorders

Advancements in cell and gene therapy and other cutting-edge technologies are enabling the development of novel therapies for diseases that have significant unmet need. This includes several rare genetic disorders for which there are few or no FDA-approved disease-modifying therapies currently available. Realizing the potential of novel therapeutic approaches in these indications may offer hope to patients but also raises a variety of challenges and questions for drug developers, regulatory agencies, clinicians, payors, and patients themselves. Perhaps the most challenging of these questions is defining what constitutes clinical benefit for patients who are all but guaranteed poor or limited outcomes with currently available interventions and natural disease progression. As cutting-edge therapies advance through clinical development there is an immediate need to answer these questions as well as develop and implement clinical endpoints that are meaningful for all stakeholders.

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