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Hyatt Regency Bethesda

2015 年 10 月 07 日 7:00 上午 - 2015 年 10 月 07 日 4:30 下午

One Bethesda Metro Center, 7400 Wisconsin Avenue, Bethesda, MD 20814-5326

Achieving Meaningful Regulatory and Clinical Outcomes for Patients: Strategies in Rare Disease Therapy Development

A collaborative workshop designed to examine practical issues in the structuring of rare diseases therapeutic development programs.

Session Chair(s)

Steven L. Roberds, PhD

Steven L. Roberds, PhD

Chief Scientific Officer

Tuberous Sclerosis Alliance, United States

Speaker(s)

Tiffany  House, JD

Addressing the Challenges of Early Patient Engagement

Tiffany House, JD

Acid Maltase Deficiency Association (AMDA), United States

President

Richard  Klein

Putting it All Together

Richard Klein

GE2P2 Global Foundation, United States

Director, Expanded Access Programs & Policy, Former FDA, Patient Liaison

Molly  White

Interactive Panel Discussion

Molly White

Myotonic Dystrophy Foundation, United States

Executive Director

Joel  Beetsch, PhD

Joel Beetsch, PhD

Celgene Corporation, United States

Vice President, Global Patient Advocacy

Tiffany  House, JD

Tiffany House, JD

Acid Maltase Deficiency Association (AMDA), United States

President

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