Speakers
2025 Speaker Submissions Now Open
Apply to be a speaker at the 2025 NORD Summit! We are seeking expert speakers who can share their perspectives on critical topics in rare disease, including but not limited to:
- Pathways to Patient Access
- Health Equity
- Patient-Centric Clinical Trials
- Drug Development
- Accelerating Diagnosis
- Medical Devices
- AI/Digital Technology Innovations in Rare Disease
All submissions must be received by 11:59 p.m. ET on March 21, 2025.
2024 Speakers
Commissioner of Food and Drugs
FDA
Robert M. Califf, M.D
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
Vice President
Federal Affairs, ALS Association
Rich Brennan, MA
Deputy Director, Division of Rare Diseases Research Innovation
NCATS, NIH
P.J. Brooks, PhD
Office of Congresswoman Anna G. Eshoo (CA-16)
Jordan Brossi
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
General Manager, US Rare Diseases
Sanofi
R. Duane Clark
VP of Corporate Relations
National Organization for Rare Disorders (NORD)
Patrick Collins
Vice President for Federal Affairs
Leukemia & Lymphoma Society
Brian Connell
Vice President of Therapeutics Development
California Institute of Regenerative Medicine
Abla Creasey, PhD
VP, Data Science
Jazz Pharmaceuticals
Mat Davis
Director, Advocacy & Engagement Strategy
Center for Rare Diseases, ICON
Kendall Davis, MPH
Director of Research, Co-founder
Malan Syndrome Foundation
Christal Delagrammatikas, PhD
Vice President, Policy
National Heath Council
Jennifer Dexter
Senior Partner, Global Public Policy, Regulatory & Governmental Strategy
Flagship Pioneering
Tom DiLenge
Senior Vice President of Policy and Advocacy
Cystic Fibrosis Foundation
Mary Dwight
Co-Founder & President
Every Cure
David Fajgenbaum, MD, MBA, Msc
Director of the Office of Clinical Evaluation (Cell and Gene Therapies)
Super Office of Therapeutics Products(OTP), CBER, FDA
Lola Fashoyin-Aje, MD, MPH
Principal and Founder, Rubrum Advising
Former CMS Chief Medical Officer and Director of the Center for Clinical Standards and Quality
Lee Fleisher, MD, LLM
Entrepreneur in Residence
Google Ventures
Nicole Gaudelli
CEO
NORD
Pamela Gavin
Chief Executive Officer
Agios Pharmaceuticals
Brian Goff
President and CEO
Blueprint Medicines
Kate Haviland
CEO
Healr Solutions, Steering Committee Member, MA RDAC
Guadalupe Hayes-Mota, MBA, MS, MPA
Research Director
Real-World Evidence (RWE), Duke-Margolis Institute for Health Policy
Rachele Hendricks-Sturrup, DHSc, MSc, MA
Senior Policy Analyst
NORD
Allison Herrity, MPH
Division of Medical Genetics
University of Washington School of Medicine
Fuki M. Hisama, MD, FACMG
Executive Vice President, Global Product Strategy & Commercialization
Biogen
Rachid Izzar
Founder
The Calliope Joy Foundation and Cure MLD
Maria Kefalas, PhD, MA
Executive Director
TBRS Community
Jill Kiernan
President, US Business Unit and US Country Head
Takeda
Julie Kim
Ropes & Gray LLP
Gregory Levine, JD
Associate Director for Innovation and Partnership
OCP, OTS, CDER, FDA
Qi Liu, PhD, MStat, FCP
Co-Founder and Executive Chairman
RallyBio
Martin Mackay, PhD
University of Rochester
Samuel Mackenzie, MD, 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
Rare Community Member
Mayra Martinez
Global Rare Disease Commercial Lead
Pfizer
Gemma Mayman
Rare Community Member
Maria Miller
President of R&D
Taysha Gene Therapies
Sukumar Nagendran, MD
Chief Medical & Scientific Officer
National Organization for Rare Disorders (NORD)
Edward Neilan, MD, PhD
CEO
TargetCancer Foundation
Jim Palma
Associate Director, Medicaid Market Access
GeneDx
Holly Paparounis
Senior Research Scientist
RTI International
Holly Peay, PhD, MS
Director of CDER Office of New Drugs
Division of Rare Disease and Medical Genetics, FDA
Catherine Pilgrim-Grayson, MD, MPH
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
Director
National Center for Advancing Translational Sciences (NCATS), NIH
Joni Rutter, PhD
National Bleeding Disorders Foundation
Maria Santaella, RN-BC, MSN, PhD(c)
Senior Vice President, Science and Technology
Medical Device Innovation Consortium
Joseph Sapiente
President
Scheer & Company
David Scheer
Senior Director, FasterCures
Milken Institute Health
Kristin Schneeman
Deputy Administrator and Director of the Center for Medicare
Centers for Medicare & Medicaid Services (CMS)
Meena Seshamani, MD, PhD
CBER Office of Therapeutic Products
Office of Clinical Evaluation
Rosa Sherafat-Kazemzadeh, MD
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
Associate Professor of McKusick-Nathans Department of Genetic Medicine, Associate Professor of Pediatrics
Johns Hopkins University
Nara Sobriera, MD, PhD
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
General Counsel
Senate HELP Committee
Barrett Tenbarge
Managing Principal
Leavitt Partners
Josh Trent
Executive Director
Rare Disease Diversity Coalition
Jenifer Waldrop, MS
Executive Vice President, Head of Clinical Development and Clinical Operations
Savara
Yasmine Wasfi, MD, PhD
Partner
Goodwin Procter LLP
Matt Wetzel
Vice President and Head of Commercial Strategy and AI
OM1
Joseph Zabinski, PhD, MEM
Partner
OrbiMed
Tal Zaks, MD, PhD
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