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

Panel:
Patient Perspectives on Gene Therapy - Patients and caregivers representing several rare disease communities share their thoughts, hopes and concerns regarding gene therapy

Sponsored by:
Center for Orphan Drug Research
University of Minnesota Medical School Stem Cell Institute

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