Duchenne Clinical Trials & Drug Development

In rare diseases such as Duchenne, where the disease population is small, the drug development process can vary from that of more common diseases, and totally new treatment approaches such as gene therapies are emerging. Therefore, it is critical that families accurately understand the process and how they can help support—and possibly benefit from—the development of treatments and cures.

– Moderator: Sharon Hesterlee, PhD, Executive VP, Portfolio Development & Head of Neuromuscular Programs, Asklepios Biopharmaceuticals (AskBio)

– Module 1: Peter Marks, MD, PhD, Director, Center for Biologics Research, Food and Drug Administration (FDA)

– Module 2: Petra Kaufmann, MD, Vice President, Research and Development, Translational Medicine, AveXis, Inc.

– Module 3: Barry Byrne, MD, PhD, Director, UF Powell Gene Therapy Center, and Associate Chair, Pediatrics, University of Florida College of Medicine

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