Vice President of Research
Institute for Clinical & Economic Review (ICER)

Deputy Director, Office of Clinical Evidence and Analysis, CDRH
US Food & Drug Administration (FDA)

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

Executive Director, Head of Patient Advocacy and External Engagement
Pfizer, Inc

Patient-Focused Drug Development Program Direction, CDER
US Food & Drug Administration (FDA)

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
National Center for Advancing Translational Sciences (NCATS), NIH

Director of our Division of Services for Children with Special Healthcare Needs
US Department of Health and Human Services' Health Resources and Services Administration (HRSA)

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

Faculty, Bioethicist-Developmental Scientist
Boston Children's Hospital and Harvard Medical School

United States Commissioner of Food & Drugs

Senior Clinical Analyst for Stakeholder Engagements, CDER
US Food & Drug Administration (FDA)

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

Rare Community Member
Peripartum Cardiomyopathy (PPCM) & Post-Transplant Lymphoproliferative Disorder (PTLD)

Rare Disease Policy Director
Little Hercules Foundation

Associate Director for Real World Evidence Analytics, Office of Medical Policy, CDER
US Food & Drug Administration (FDA)

Executive Director
International Fibrodysplasia Ossificans Progressiva Association (IFOPA)

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

Director of Membership
National Organization for Rare Disorders (NORD)

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)

Senior Managing Director
Manatt Health

Director of Corporate Development
Cholangiocarcinoma Foundation

Executive Director
Hemophilia Foundation of Southern California

Global Head of Rare Disease Medical Affairs
Sanofi

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

Program Director, Division of Clinical Research
National Institute of Neurological Disorders & Stroke, NIH

Senior Project Manager
Critical Path Institute

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

Pediatric Team Leader, CDER
US Food & Drug Administration (FDA)

Rare Community Member

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

Professor of Pediatrics
Duke University School of Medicine

Chief Medical Officer
Entrada Therapeutics

President & CEO
Platelet Disorder Support Association (PDSA)

Founder & Chief Executive Officer
Alliance to Cure Cavernous Malformation

Clinical Psychologist
Leidos Biomedical Research, Inc./National Cancer Institute

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

Chief Executive Officer
Foundation for Sarcoidosis Research

Deputy Director of Science, Office of Pediatric Therapeutics, Office of the Commissioner
US Food & Drug Administration (FDA)

Head of US Regulatory Affairs, Global Rare Diseases
Chiesi USA Inc

Vice President Public Policy & Advocacy
Muscular Dystrophy Association

Program Director
National Institute of Neurological Disorders and Stroke, NIH

Executive Director, Rare Disease COA Consortium
Critical Path Institute

Assistant Professor of Clinical Medical and Molecular Genetics
Indiana University

President & CEO
Indian Organization for Rare Diseases

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)

Medical Director, Children's National Rare Disease Institute
Children's National Hospital

Director of Community Outreach
Charcot-Marie-Tooth Association

Associate Director of Pharmacoepidemiology
Oncology Center of Excellence, US Food & Drug Administration (FDA)

Director of Federal Policy
National Association of Medicaid Directors

Clinical Science Program Manager
Fanconi Anemia Research Fund

President & CEO
TSC Alliance

Executive Director
National Ataxia Foundation

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)

President & Chief Executive Officer
National Organization for Rare Disorders (NORD)

President & Chief Executive Officer
Alpha-1 Foundation

Vice President of Community Engagement
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

Chief of Staff, Office of the Commissioner
US Food & Drug Administration (FDA)

Branch Chief and Senior Investigator
National Heart, Lung, and Blood Institute, NIH

Senior Vice President and Head of Early Clinical
Denali Therapeutics

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

President
United Porphyrias Association

Vice President, Director Patient Centered Research
Myositis Support & Understanding

Chief Executive Officer
Youth Buddies

Senior Medical Director, Rare Diseases
Takeda

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

Office of Pediatric Therapeutics (PTC)
US Food & Drug Administration (FDA)