ARM's Rare Disease Roundtable
This event is the first in our series of patient roundtables in which ARM will partner with leading patient advocacy organizations to provide opportunities for patient advocates to meet with leaders in academia, government and industry. The goals of the event are to educate multi-sector stakeholders about the latest scientific progress and the promise that gene therapy, cell therapy and other regenerative medicines offer, helping to bridge the gap between industry and patient groups who are working toward the common goal of durable, and potentially curative, therapies.
Washington, DC

Rare Disease

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.

Karen Kozarsky, Ph.D., CEO, Vector Partners

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

Ron Bartek, President & Co-Founder, Friedreich’s Ataxia Research Alliance

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