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Exon Skipping and Read Through Agents Panel
A brief introduction on the rationale behind mechanisms of endogenous dystrophin restoration, like exon skipping and read through agent therapies, followed by critical information and updates from the companies in this space. Moderated discussion included.
Barry Byrne, MD, PhD
Professor and Associate Chair
University of Florida, Department of Pediatrics
Brett Crawford, PhD
Executive Director and Head of the Musculoskeletal Therapeutic Area
BioMarin Pharmaceutical Inc.
Mark Stahl, MD, PhD
Senior Medical Director, Clinical Development
Avidity Biosciences
Deanna Tucker, PharmD
Senior Medical Science Liaison
Sarepta Therapeutics
Video
