Speakers

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2026 Expert Speakers

When you join us for the NORD Rare Disease Scientific Symposium, you’ll engage with – and learn from – world-class rare disease researchers and experts. From academia and clinical settings to industry and regulatory bodies, our speakers will share cutting-edge research and inspire new collaboration.

Teresa Buracchio, MD

Director, Office of Neuroscience

CDER, FDA

Sarah Fuchs, MD, MSCI

Medical Officer

CDER, FDA

Paul Ayoub, PhD, MBA

Founder & Chief Executive Officer

Rarity PBC

Annette Bakker

CEO

Children’s Tumor Foundation

Jaishri Blakeley, MD

Professor of Neurology, Neurosurgery and Oncology

Johns Hopkins University

NORD® Rare Disease Center of Excellence

Teresa Buracchio

Director, Office of Neuroscience

CDER, FDA

C. Lee Cohen, MD, MBA

Medical Officer

CDER, FDA

Amy Comstock Rick, JD

Director of Strategic Coalitions

FDA Rare Disease Innovation Hub

Gabrielle Conecker, MPH

Executive Director & Co-Founder

Decoding Developmental Epilepsies

Gerald Cox, MD, PhD, FACMG

Consultant, Gerald Cox Rare Consulting, LLC; Assistant in Medicine, Division of Genetics and Genomics, Staff Physician in Genetics, Part-time, Boston Children's Hospital

NORD® Rare Disease Center of Excellence

Oxana V. Crysler, MD, MHS

Clinical Associate Professor of Internal Medicine

University of Michigan, Rogel Cancer Center

NORD® Rare Disease Center of Excellence

Amena Fine

Assistant Professor of Neurology

Kennedy Krieger Institute

NORD® Rare Disease Center of Excellence

Pamela Gavin

CEO

NORD

Tracy Beth Høeg, MD, PhD

Acting Director

Center for Drug Evaluation and Research (CDER), FDA

Michael Hund, MBA

CEO

EB Research Partnership

Yuan Ji, PhD

Professor of Biostatistics

University of Chicago

Kelley Kidwell, PhD

Professor of Biostatistics, Interim Associate Dean of Faculty Affairs

University of Michigan School of Public Health

NORD® Rare Disease Center of Excellence

Margarita L. Valdez Martínez

Chief Advocacy Officer

ASGCT

Amy Palmer Laster, PhD

Chief Scientific Officer

Foundation Fighting Blindness

Savannah Latimer

Clinical Research Assistant, Division of Emergency Medicine

Children’s National Hospital

NORD® Rare Disease Center of Excellence

Maurice Leary

Children’s National Hospital

NORD® Rare Disease Center of Excellence

Wonyul Lee, PhD

Senior Statistical Reviewer

CDER, FDA

Craig Lipset

Co-Chair; Clinical Innovation

Decentralized Trials & Research Alliance; Buffalo Initiative

David Margolin, MD, PhD

VP of Clinical Development

uniQure

Michelle Mellion, MD

Chief Medical Officer, Clinical Development and Strategy Advisor

Former CMO; EveryONE Medicines

Olivia Morgan, PhD

Statistician

FDA

Kasha Morris, M.Ed

Co-Founder

TANGO2 Research Foundation

Amy Nicole Nayar, PharmD, MS

Vice President, Medical Affairs

Lundbeck

Edward Neilan, MD, PhD

Chief Medical and Scientific Officer

NORD

Gwen Nichols, MD, EVP

Chief Medical Officer

Blood Cancer United

Mayowa Osundiji, MBBS, PhD

Medical Geneticist

Mayo Clinic

NORD® Rare Disease Center of Excellence

Ravi Pathak, PhD, MBA

Medical Director, Lysosomal Storage Disorders, US Medical

Takeda

Nicole D. Paulk, PhD

CEO, Founder and President

Siren Biotechnology

Terry Pirovolakis

Founder & CEO

Elpida Therapeutics

Matthew Porteus, MD, PhD

Professor of Pediatrics

Stanford University

NORD® Rare Disease Center of Excellence

Srilakshmi Raj, PhD

Assistant Professor of Genetics

Albert Einstein College of Medicine

NORD® Rare Disease Center of Excellence

Amy Raymond, PhD, PMP

Executive Director, Therapeutic Strategy Lead, Rare Disease & Cellular and Genetic Medicines

Worldwide Clinical Trials

Matthew S. McCoy, PhD

Assistant Professor of Medical Ethics and Health Policy

University of Pennsylvania, Perelman School of Medicine

NORD® Rare Disease Center of Excellence

Lowell Schiller, JD

Nonresident Senior Scholar; former Principal Associate Commissioner for Policy

University of Southern California Schaeffer Center; FDA

NORD® Rare Disease Center of Excellence

Natasha Shur, MD

Medical Geneticist, Professor of Pediatrics

Children’s National Hospital

NORD® Rare Disease Center of Excellence

Tracey Sikora

Vice President of Research and Clinical Programs

NORD

Judy Stecker

Founder, Wheeler’s Warriors; Patient Advocate, former HHS Deputy Chief of Staff

Heather Stone, MPH

Health Science Policy Analyst

FDA

Theresa Strong, PhD

Director of Research Programs

Foundation for Prader-Willi Research

Mark Trusheim, MSm

Senior Strategy Advisor, NEWDIGS

Tufts Medical Center

Julia Vitarello

Founder & CEO

Mila's Miracle Foundation

Angela Waanders, MD, MPH, MS

Section Head, Neuro-Oncology

Ann and Robert H. Lurie Children’s Hospital of Chicago

NORD® Rare Disease Center of Excellence

Annica Wayman, PhD

Deputy Director

National Center for Advancing Translational Sciences, NIH

Danny Yeh, PhD

Vice President, Rare Disease, Center of Excellence

AESARA

Timothy Yu, MD, PhD

Associate Professor of Pediatrics

Harvard Medical School, Boston Children’s Hospital

NORD® Rare Disease Center of Excellence

Susana Zaph, PhD

Senior Director, Head of Quantitative Systems Pharmacology -US

Sanofi