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AAN 2021 | Gene therapy for Duchenne muscular dystrophy (DMD)

Jerry Mendell, MD, FAAN, Nationwide Children’s Hospital & Ohio State University, Columbus, OH, comments on the use of gene therapy in patients with Duchenne muscular dystrophy (DMD). Prof. Mendell group is working on this disease, applying what was learned from spinal muscular atrophy (SMA) type I studies. The challenges in treating DMD compared to SMA, the treatment principles, and the expected outcomes are discussed. This interview took place during the American Academy of Neurology (AAN) 2021 Annual Meeting.


Prof. Mendell reports the following disclosures:
Consultant for Sarepta therapeutics, Novartis Inc, Vertex Inc