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ARM's Rare Disease Roundtable

January 27, 2016

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