Gene Therapy for DMD How it Works and the Ongoing Clinical Trials

Presenter:
Perry Shieh, MD, PhD. Professor of Neurology and Pediatrics, University of California Los Angeles.
Held on August 23 and 24, 2022, this MDA Engage DMD Symposium empowers individuals and families with knowledge and resources specific to Duchenne muscular dystrophy (DMD). Information is provided by experts in the field on topics such as research, clinical trials, drug development, adaptive equipment and technology and home modifications. Members of the DMD community also discuss and share experiences around self-advocacy.
MDA would like to thank our 2022 DMD Symposium supporters: BioMarin Pharmaceutical, Edgewise Therapeutics, NS Pharma, PTC Therapeutics, Sarepta Therapeutics, Entrada Therapeutics, and REGENXBIO.