Agenda:

1)      Welcome, Goals for the Workshop Series, Overview

• Cara M. Weismann, PhD, Orphan Disease Center, University of Pennsylvania, Philadelphia, PA

2)     “Lessons Learned from the GM1 Natural History Data Sharing Collaborative Effort"

• Kathleen Kirby, Advisor to Cure GM1, Drug Development Consultant, Viridian Strategies, Boston, MA

3)    “GLIA-CTN: Challenges and Opportunities from the Perspective of a Multidisciplinary Rare Disease Program”

• Adeline Vanderver, MD, Program Director of the Leukodystrophy Center of Excellence, Children’s Hospital of Philadelphia, Jacob A. Kamens Endowed Chair in Neurological Disorders and Translational NeuroTherapeutics, Professor of Neurology and Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA

4)  “Vivli and Critical Path, Sharing Rare Disease Data”

• Julie Wood, Chief Operating Officer, Vivli, Cambridge, MA

• Alexandre Bétourné, Ph.D., Pharm.D., Executive Director, Rare Disease Cures Accelerator – Data Analytics Platform (RDCA-DAP), Tucson, AZ, USA

  • Presentation slides

5)      Questions & Discussion

• Presenters panel: Kathleen Kirby, Adeline Vanderver, MD, Julie Wood, Alexandre Bétourné, Ph.D., Pharm.D., & Cara M. Weismann, PhD

6)      Summary, Wrap up, Future Directions

• Cara M. Weismann, PhD, Orphan Disease Center, University of Pennsylvania, Philadelphia, PA