Associate Director of Advocacy
NORD

GFPD Diversity Committee Board Member, Sibling Caregiver and Advocate

Director National Center for Advancing Translational Sciences
National Institutes of Health

Director
US Senate HELP Committee

American Porphyria Foundation

Professor, Division of Genetics and Metabolism Departments of Pediatrics and Genetics, Cell Biology & Development
University of Minnesota

Associate Director for Patient Outcomes
Oncology Center of Excellence, FDA

Senior Vice President, Access and External Affairs
Genentech

Director of Research Programs
NORD

Associate Director of Research Programs
NORD

Executive Director
Gorlin Syndrome Alliance

VIce President, Global Commercial Development, Rare Disease
Pfizer Biopharmaceuticals Group

Co- Founder and Executive Director
The Global Foundation for Peroxisomal Disorders

Associate Professor of Medicine, Vice Chair for Diversity and Inclusion Director, Nephrology Fellowship Program
Icahn School of Medice at Mount Sinai

Deputy Director for Operations
Center for Drug Evaluation and Research(CDER) , Food and Drug Administration

White House Presidential Innovation Fellow, Executive Office of the Presidents
Office of Science and Technology Policy

Director of Membership
NORD

CEO
Retrophin

Deputy Director, Office of Medical Policy - Center for Drug Evaluation and Research
Food & Drug Administration

Co-founder and Executive Director of the Castleman Disease Collaborative Network (CDCN), Assistant Professor of Medicine in Translational Medicine & Human Genetics at the University of Pennsylvania, and Associate Director, Patient Impact for the Penn Orphan Disease Center

Chief Strategy Officer
NORD

Professor of Pediatirics Medical Director, Kidney & Liver Tumor Programs Director, Advanced Cancer Therapeutics Network Director, Pilot Translational and Clinical Studies Program, CCTST
Cincinnati Children's Hospital Medical Center, Division of Oncology

Head, U.S. and Sanofi Genzyme Corporate Affairs
Sanofi Genzyme

President & Chief Executive Officer
Black Women's Health Imperative

Managing Director
Sanofi Ventures

Commissioner
US Food and Drug Administration

Associate Director, Quantitative Patient Inputs & RWE
Food and Drug Administration

Critical Path Institiute, NORD Board Member

Director, Center for Innovation in Global Health, Professor of Medicine, Georgetown University School of Medicine, and Distinguished Scholar and Program Director, O'Neill Institute for National and Global Health Law
Georgetown University

President
Fibrolamellar Cancer Foundation

Senior Director of Health Professional Programs
Aplastic Anemia & MDS International Foundation

President & Founder
Foundation 29

Acting Director
Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine at FDA

General Partner
Sofinnova Investments

Senior Genomic Consultant
GeneDx

Representative D- IL 2nd Disctrict
State of Illinois

Director
National Institute of Neurological Disorders and Stroke

Group Vice President, Oncology Targeted Therapeutics
Incyte

Associate Director, DCEPT/OTAT Center for Biologics Evaluation and Research
Food and Drug Administration

Executive Director
Duchenne Regulatory Science Consortium, Rare Diseases Cures Accelerator- Data and Analytics Platform

MSc, MCC, MMBA, Chief Patient Officer
Leo Pharma

Chief Executive Officer
EURORDIS

Deputy Staff Director
House Committee on Energy & Commerce

President & Chief Executive Officer
Global Blood Therapeutics

Co-Founder
Rallybio

Chief Executive Officer
Mito ACTION

Director
CBER FDA

State Representative, NH

Director, Office of Orphan Products Development
Food and Drug Administration

President and Chief Executive Officer
Biotechnology Innovation Organization (BIO)

Board of Directors
CureSMA

Founder, Chief Executive Officer
Hillstream BioParma

Practice Director, Center for Healthcare Transformation
Avalere Health

Member, Board of Directors
NephCure Kidney Institute

Children's Hospital of Pittsburgh (CHOP)

Chairman
Acceleron

President & CEO
Alpha-1 Foundation

Associate Director of Operations and Scientific Outreach for the Broad Cancer Program, Associate Director of Count Me In
Broad Institute

Executive Director
TargetCancer Foundation

Founder & CEO
The Mighty

Management Analyst, Office of Rare Diseases Research (ORDR)
National Center for Advancing Translational Sciences (NCATS)

Health Policy Advisor
US Senate Committee on Finance

Senior Policy Advisor on Budget and Health to Congresswoman Nancy Pelosi (D-CA)

Childrens National and the Rare Disease Institute

Director
Rate Tumor Initiative, CCR, NCI

Food & Drug Administration

Director Of Policy
NORD

President and CEO
NORD

Vice President for Research and Chief Scientific Officer
CHOC Children's Hospital of Orange County

Director of Strategic Planning & Project Development
NORD

Scheer & Company Inc

Senior Research Programs Manager
NORD

President/Executive Director
ADNP Kids Research Foundation

Founder & CEO
Applied Therapeutics

Sr. Scientific Director, Global Medical Affairs
Novartis Gene Therapies

Vice President, Policy and Regulatory Affairs
NORD

Eversana

Cystic Fibrosis Foundation

Global Medical Lead, Hemophilia
BioMarin Pharmaceutical Inc

Associate Professor in Psychiatry, Neurology and Pediatrics NORD Board Member
UCLA David Geffen School of Medicine Los Angeles, CA

Health Science Policy Analyst
Food & Drug Administration

Director of ResearchPrograms
Foundation for Prader-Willi Research

Professor of Pediatrics, Wallace Chair of Pediatrics Division of Allergy Immunology
CHOP (Children's Hopsital of Philadelphia)

Division Chief, Genetics and Metabolism, Director, Rare Disease Institute
Children's National

Wisconsin Rare Action Network Volunteer State Ambassador
NORD

Assistant Professor, Director VIDE Program Horae Gene Therapy Center, Department of Microbiology and Physiological Systems
University of Massachusetts Medical School

Rare Action Network, NORD

Chief Executive Officer
Board Veritas

Director of Research
Association for Creatine Deficiencies

Director, Clinical Trial Services Director, Ocular & Rare Disease Genetics Services
InfomedDNA

Head, Organic Acid Research Section Senior Investigator, National Human Genome Research Institute
National Institutes of Health

Executive Director
Dup15q Alliance

Senior Director Clinical Development
Foundation Medicine Inc.

Deputy Director National Institute on Minority Healh and Health Disparities
National Institutes of Health

President and CEO
Canadian Organization for Rare Disorders

Executive Director
International Pemphigus and Pemphigoid Foundation (IPPF)

VP of Biological Development
Editas Medicine