Rare Disease Day 2019
Clinical Gene Therapy: The Future is Now
Thank you for all who joined us!
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.
Watch the Presentations Now
Rare Disease Day 2019
Program
Senator Amy Klobuchar
Senator Tina Smith
Co-Sponsors of Rare Disease Day
Center for Orphan Drug Research
University of Minnesota Medical School Stem Cell Institute
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.