CLINICAL & PATIENT EDUCATION ROUNDTABLE
Rare Disease
IN PARTNERSHIP WITH:
Friedreich’s Ataxia Research Alliance
Program Agenda
2:00-2:15pm | Welcome & Introductory Remarks
Ron Bartek, President & Co-Founder, Friedreich’s Ataxia Research Alliance
Morrie Ruffin, Managing Director, Alliance for Regenerative Medicine
2:15-2:45pm | Featured Talk
Barry Byrne, M.D., Ph.D., Director, Powell Center for Rare Disease Research and Therapy, University of Florida
2:45-3:45pm | Research for Rare Disease: Unique Opportunities & Challenges
A multi-stakeholder panel discussion emphasizing the unique opportunities & challenges facing research and development in rare and orphan diseases while exploring the regenerative medicine landscape for rare diseases, basic research in the field and the company decision-making process.
Moderator:
Karen Kozarsky, Ph.D., CEO, Vector Partners
Speakers:
Deborah Ascheim, M.D., Chief Medical Officer, Capricor Therapeutics
Julie Venners Christensen, Head of Global Patient Advocacy – Gene Therapy, R&D, Rare Disease Unit, GlaxoSmithKline
Sharon Hesterlee, Ph.D., Chief Science Officer, Myotonic Dystrophy Foundation
3:45-4:00pm | Break
4:00-5:00pm | Collaborating on Cures for Rare Disease
Case studies on successful partnerships between advocacy organizations and ARM companies
Moderator:
Ron Bartek, President & Co-Founder, Friedreich’s Ataxia Research Alliance
Speakers:
Jennifer Cutie, M.D., Associate Director, Patient Advocacy, Voyager Therapeutics
Tim Miller, Ph.D., President & CEO, Abeona Therapeutics
Barbara Wuebbels, Vice President of Patient Advocacy, Audentes Therapeutics
5:00-5:30pm | Featured Talk: The Patient Perspective
Michele Rhee, Head of Global Patient Affairs, bluebird bio
Jean Walsh, Patient Advocate; Ambassador, Friedreich’s Ataxia Research Alliance
5:30-5:45pm | Closing Remarks: Next Steps
Michael Werner, Executive Director, Alliance for Regenerative Medicine
5:45-7:00pm | Networking & Cocktail Reception
For more information:
Chelsey Hathaway
Manager, Patient Advocacy & Events
chathaway@alliancerm.org
