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)