A New Era of Rare Disease Diagnostics and Therapeutics

James M. Wilson, MD, PhD

Rose H. Weiss Professor and Director, Orphan Disease Center,

Perelman School of Medicine, University of Pennsylvania

George Weiss

Founding Donor, Orphan Disease Center

Jonathan A. Epstein, MD

Executive Vice Dean and Chief Scientific Officer,

Perelman School of Medicine, University of Pennsylvania

Francis S. Collins, MD, PhD

Director, National Institutes of Health

Holly Fernandez Lynch, JD, MBE

Assistant Professor of Medical Ethics and Health Policy,

Perelman School of Medicine, University of Pennsylvania

Alison Bateman-House, PhD, MPH, MA

Assistant Professor, Department of Population Health,

NYU Grossman School of Medicine

Jeremy Snyder, PhD

Professor of Health Sciences, Simon Fraser University

Timothy Yu, MD, PhD

Associate Professor of Pediatrics, Harvard Medical School

Jayne Gershkowitz

Chief Patient Advocate, Amicus Therapeutics

Lynn Barghout Jafar

Co-Founder, Loulou Foundation

Glen N. Gaulton, PhD

Vice Dean and Faculty Director, Center for Global Health

Professor of Pathology and Laboratory Medicine,

Perelman School of Medicine, University of Pennsylvania

Luiz Henrique Mandetta, MD

Former Minister of Health Brazil

Khaled Otaifi, MD

Director of the Metabolic Disease Service at the

Egyptian Ministry of Health

Joan Benson, MD, MPH

Executive Director of Global Health Partnerships, Merck

J. Larry Jameson, MD, PhD

Executive Vice President, University of Pennsylvania

for the Health System; Dean, Perelman School of Medicine

Majid Jafar

Co-Founder, Loulou Foundation

Tachi Yamada, MD

Former President of R&D, GSK and Takeda

David Cameron

Former UK Prime Minister

Pat Furlong

Founding President and CEO,

Parent Project Muscular Dystrophy (PPMD)

Alicyn Campbell, MPH

Head of Digital Health, Oncology R&D, AstraZeneca

David Fajgenbaum, MD, MBA, MSc

Assistant Professor of Medicine, Perelman School of Medicine,

University of Pennsylvania

Founder, Castleman Disease Collaborative Network

Betsy Bogard, MS

Head of Program and Alliance Management

5 AM Ventures, 4:59 Initiative

Emma A. Meagher, MD

Vice Dean and Chief Clinical Research Officer

Perelman School of Medicine, University of Pennsylvania

Janet Woodcock, MD

Acting Commissioner, US Food & Drug Administration

Annalisa Jenkins, MBBS, FRCP

Biotech Executive

John Crowley, JD, MBA

CEO, Amicus

Emil Kakkis, MD, PhD

CEO, Ultragenyx

Daniel J. Rader, MD

Seymour Gray Professor of Molecular Medicine, Perelman School ofMedicine, University of Pennsylvania

Chair, Department of Genetics

Michael S. Brown, MD

Regental Professor, Molecular Genetics and Internal Medicine

UT Southwestern

James M. Wilson, MD, PhD

Rose H. Weiss Professor and Director, Orphan Disease Center,

Perelman School of Medicine, University of Pennsylvania