2024 Speakers
More speakers to be announced weekly
Rare Community Member
Tanita Allen
Professor, Division of Genetics & Metabolism, University of Wisconsin School of Medicine and Public Health; Director of Newborn Screening Program at Wisconsin State Laboratory of Hygiene
Mei Baker, MD, FACMG
CEO
American Society of Gene and Cell Therapy
David Barrett, JD
Rare Community Member and Associate Professor
Department of Nutrition and Food Studies, College for Community Health, Montclair State University
Mousumi Bose, PhD
Deputy Director, Division of Rare Diseases Research Innovation
NCATS, NIH
P.J. Brooks, PhD
Director, Division of Rare Diseases Research Innovation
NIH
Dominique C. Pichard, MD, MS
Director, Access Policy
Muscular Dystrophy Association
Joel Cartner, Esq.
Center Director for the Center for Drug Evaluation and Research (CDER)
Food and Drug Administration
Patrizia Cavazzoni, M.D.
Head of Global Rare Disease and Member of the Board
Chiesi Pharmaceuticals
Giacomo Chiesi
VP, Data Science
Jazz Pharmaceuticals
Mat Davis
Senior Vice President of Policy and Advocacy
Cystic Fibrosis Foundation
Mary Dwight
Co-Founder & President
Every Cure
David Fajgenbaum, MD, MBA, Msc
Entrepreneur in Residence
Google Ventures
Nicole Gaudelli
Chief Executive Officer
Agios Pharmaceuticals
Brian Goff
President and CEO
Blueprint Medicines
Kate Haviland
Research Director
Real-World Evidence (RWE), Duke-Margolis Institute for Health Policy
Rachele Hendricks-Sturrup, DHSc, MSc, MA
Executive Vice President, Global Product Strategy & Commercialization
Biogen
Rachid Izzar
President, US Business Unit and US Country Head
Takeda
Julie Kim
Co-Founder and Executive Chairman
RallyBio
Martin Mackay, PhD
Director, Center for Biologics Evaluation and Research (CBER)
US Food & Drug Administration (FDA)
Peter Marks, MD, PhD
Chief Medical Officer
FDA
Hilary Marston, MD, MPH
President of R&D
Taysha Gene Therapies
Sukumar Nagendran, MD
Senior Research Scientist
RTI International
Holly Peay, PhD, MS
Executive Partner
Sofinnova Investments
Maha Radhakrishnan, MD
Vice President, Policy & Regulatory Affairs
National Organization for Rare Disorders (NORD)
Heidi Ross, MPH
Science Program Director
CSNK2A1 Foundation
Gabrielle Rushing, PhD
President
Scheer & Company
David Scheer
Chief of Staff and Strategy
Foundation for Sarcoidosis Research
Tricha Shivas
Senior Medical Advisor, Real-World Evidence Analytics, Office of Medical Policy
CDER, FDA
Kimberly Smith, MD, MS
CEO
Center for Innovation & Value Research
Jason Spangler MD, MPH, FACPM
Interim Director, Center for Devices and Radiological Health (CDRH)
US Food & Drug Administration (FDA)
Michelle Tarver, MD, PhD
Managing Principal
Leavitt Partners
Josh Trent
Partner
Goodwin Procter LLP
Matt Wetzel
Partner
OrbiMed
Tal Zaks, MD, PhD
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