Rare Disease Day 2019 - Clinical Gene Therapy: The Future is Now

This year’s program held on March 1, 2019, furthered our understanding of gene therapy by hearing from a clinician and a panel of patients and parents. The clinician provided an overview of current therapies and therapies that are in the pipeline, whereas the panel featured gene therapy patients and parents of children who are potential recipients. All were welcomed to this free event. Keynote: James M. Wilson, MD, PhD Rose H. Weiss Professor and Director Orphan Disease Center, University of Pennsylvania