Deputy Director Division of Pediatric & Maternal Health, Office of New Drugs, CDER
US Food & Drug Administration (FDA)

Rare Community Member

Rare Community Member

CSO and Head of Rare Diseases
Acadia Pharmaceuticals

Senior Program Manager, Science in Society, and Lead, Rare As One Network
Chan Zuckerberg Initiative

Chief Science Officer
Kabuki Syndrome Foundation

Office of Strategic Partnerships & Technology Innovation, CDRH
US Food & Drug Administration (FDA)

Associate Chief of Genetics & Genomics
Boston Children's Hospital

Acting Director, Division of Rare Diseases Research Innovation, National Center for Advnacing Translational Sciences (NCATS)
NIH

Chief Commercial Officer
Solaxa

Commissioner
US Food & Drug Administration (FDA)

Founder and Director
Neurology Rare Disease Center

Director, Center for Drug Evaluation & Research (CDER)
US Food & Drug Administration (FDA)

Geneticist & Metabolist
Children's National Hospital

Senior Vice President, Research & Product Development
Alexion/AstraZeneca Rare Disease

Executive Director, Head of Global R&D and Regulatory Policy
BioMarin Pharmaceutical Inc.

Chief of Pediatrics
Boston Children's Hospital

Vice President, Government Relations
Alliance for Regenerative Medicine

General Manager, US Rare Diseases
Sanofi

VP of Corporate Relations
National Organization for Rare Disorders (NORD)

Director, Patient Advocacy
Horizon Therapeutics

President & Chief Executive Officer
Travere Therapeuticcs

Chief Scientific Officer
The FamilieSCN2A Foundation

Director of Research Data and Analytics
National Organization for Rare Disorders (NORD)

N/A
N/A

Psychologist
Freedman Counseling Associates

Office of Neuroscience, Office of New Drugs, CDER
FDA

Founder and Chief Innovation Officer
Deep Genomics

Chief Medical Officer
Children's Tumor Foundation

Scientist
Rogers Behavioral Health

Medical Director
Progeria Research Foundation

Director, Office of Pediatric Therapeutics, OCPP, OC
FDA

Vice President, Head, GRA Global Product Strategy
CSL Behring

Director, Policy and Regulatory Affairs
National Organization for Rare Disorders (NORD)

Vice President of the Rare and Orphan Disease Programs
Critial Path Institute

Principal
ATI Advisory

Chief Technology Officer
GE Healthcare

SVP, Chief Medical Officer
Reata Pharmaceuticals

Senior Vice President for Healthcare Policy & Research
Biotechnology Innovation Organization (BIO)

Investigator, Professor
Broad Institute; Harvard University

Professor of Pediatrics & Chief Division of Medical Genetics
University of Utah, Spencer Fox Eccles School of Medicine

Clinical Health Psychologist, Professor of Pediatrics
Children's National Hospital

Co-Founder and Executive Chairman
Rallybio

Director, Center for Biologics Evaluation and Research (CBER)
US Food & Drug Administration (FDA)

Chief of Staff, Center for Medicare
Center for Medicare and Medicaid Services (CMS)

Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine; OND, CDER
FDA

Vice President, Decentralized Clinical Trials (DCT)
Medidata

CEO
To Cure A Rose Foundation

Chief Medical Officer
Orchard Therapeutics

CEO & Founder
Folia Health

Head of US Rare Diseases
UCB

Director, Orphan Products Grants Program, OOPD
US Food & Drug Administration (FDA)

Chief Medical & Scientific Officer
National Organization for Rare Disorders (NORD)

Chief Global Development Officer
The Marfan Foundation

Rare Disorder Nurse Navigator
Washington University School of Medicine, St. Louis

Head of Health Equity Alliances & Partnerships, Center for Health Equity & Patient Affairs
Takeda

President & Chief Operating Officer
Rocket Pharmaceuticals

Chief Medical Officer and Director, Pediatrics & Special Populations, Centers for Devices and Radiological Health
US Food & Drug Administration (FDA)

President & Chief Executive Officer
Sumitomo Pharma America

Director, Office of Orphan Products Development (OOPD)
US Food & Drug Administration (FDA)

Chief Executive Officer
OrphAI Therapeutics

Founder
KIF1A.ORG

Vice President, Policy & Regulatory Affairs
National Organization for Rare Disorders (NORD)

Global Chairman, Healthcare Investment Banking
JP Morgan

Vice President, External Affairs
Genentech

President
Scheer & Company

Senior Health Policy Advisor
Senate Committee on Finance

Director, Center for Devices & Radiological Health
US Food & Drug Administration (FDA)

Program Officer
NIH, NCATS, Division of Rare Diseases Research Innovation

Co-Founder
EveryCure

Chief Medical Officer
Beam Therapeutics

Associate Professor
Johns Hopkins University

Director, Office of Strategy & Alliances Pediatric Translational Neuroscience Initiative
St. Jude Children's Research Hospital

Office of Medical Policy, CBER
Food & Drug Administration

Deputy Center Director for Transformation, CDRH
US Food & Drug Administration (FDA)

Co-Founder and Chief Executive Officer
Rallybio

Senior Director (Digital Health & Technology)
Medical Device Innovation Consortium (MDIC)

Vice President, Philanthropy, and Executive Director of the Deerfield Foundation
Deerfield

Director, Office of Therapeutic Products, CBER
FDA

President
United Porphyrias Association

Deputy Director, Center for Biologics Evaluation & Research (CBER)
US Food & Drug Administration (FDA)

Office of Clinical Evaluation, Office of Therapeutic Products , CBER
US Food & Drug Administration (FDA)

Division of Rare Diseases & Medical Genetics, Deputy Director, Office of New Drugs, CDER
US Food & Drug Administration (FDA)

Venture Partner
OrbiMEd