Executive Director
Hermansky-Pudlak Syndrome Network

Assistant Professor, Division of Medical Ethics
NYU Grossman School of Medicine

Senior Director, Federal Advocacy
American Academy of Pediatrics

RDCA-DAP Scientific Director
Critical Path Institute

Executive Director of Strategic Partnerships
Amyloidosis Research Consortium (ARC)

Program Director, US Regulatory Policy
Genentech

Acting Director, Division of Rare Diseases Research Innovation
NIH NCATS

Director, Office of Tissues and Advanced Therapies, CBER
US Food & Drug Administration (FDA)

Faculty, Bioethicist-Developmental Scientist
Boston Children's Hospital

Director
Center for Drug Evaluation & Research (FDA)

Rare Community Member
Lymphoproliferative Disorder (PTLD)

Executive Director
International Fibrodysplasia Ossificans Progressiva Association (IFOPA)

Director, Division of Rare Diseases and Medical Genetics
US Food & Drug Administration (FDA)

CEO & President
The E.WE Foundation

Associate Director, Oncology Center of Excellence; Deputy Director, Division of Oncology III- CDER/OND/OOD
Food and Drug Administration (FDA)

Branch Chief, General Medicine 1, Office of Tissues and Advanced Therapies, CDER
US Food & Drug Administration (FDA)

Co-Chair/President
NORD Rare Cancer Coalition/Fibrolamellar Cancer Foundation

President
Project GUARDIAN

Vice President of Medical and Scientific Strategy, Head of the Rare Disease Consortium
Syneos Health

Rare Community Member & Founder
Living with Holt-Oram Syndrome

Reviewer, Division of Clinical Outcome Assessment, CDER
US Food & Drug Administration (FDA)

Founder & Chief Executive Officer
Alliance to Cure Cavernous Malformation

Clinical Psychologist
National Cancer Institute

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

Program Director
National Institute of Neurological Disorders and Stroke

Executive Director, Rare Disease COA Consortium
Critical Path Institute

Assistant Professor of Clinical Medical and Molecular Genetics
Indiana University

Chief Regulatory Affairs Officer
Bridgebio Pharma

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

Executive Director
TargetCancer Foundation

Rare Community Member
MTHFS Gene Mutation

Chief Medical Officer & Director for Pediatrics & Special Populations, CDRH
US Food & Drug Administration (FDA)

Vice President of Patient Services
National Organization for Rare Disorders (NORD)

President & CEO
TSC Alliance

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

Associate Director for Policy, Office of Tissues and Advanced Therapies, CBER
US Food & Drug Administratioin (FDA)

Senior Research Programs Manager
National Organization for Rare Disorders (NORD)

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

Director, Adult Medical Genetics Program
University of Colorado, Anschutz Medical Campus

Director of Patient Education
Neuro Endocrine Tumor Research Foundation

Senior Investigator and Director
National Center for Advancing Translational Science (NCATS), NIH

Chief of Genetic and Genomic Medicine, Director of the Center for Rare Disease Therapy
UPMC Childrens Hospital of Pittsburg

President
United Porphyrias Association

Chief Executive Officer
Youth Buddies

Senior Mathematical Statistician, CBER
US Food & Drug Administratin (FDA)