April 14 – April 15, 2026
Arlington, VA

Speakers

2026 speakers will be announced soon

2025 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.

Kristina an Haack, MD

Senior Global Project Head Lysosomal Storage Disorders and Neuromuscular Diseases

Sanofi

Annette Bakker, PhD

Chief Executive Officer

Children’s Tumor Foundation

Steven Bedrick, PhD

Associate Professor of Medical Informatics and Clinical Epidemiology

Oregon Health & Science University

Susan A. Berry, MD

Professor of Genetics and Metabolism, Department of Pediatrics

University of Minnesota

NORD® Rare Disease Center of Excellence

Nancy Bolous, MD, MA, MSc

Senior Research Scientist

St. Jude Children’s Research Hospital

P.J. Brooks, PhD

Deputy Dir, DRDRI

NCATS, National Institutes of Health

Manish Butte, MD, PhD

Professor and Division Chief, Immunology, Allergy, and Rheumatology

University of California, Los Angeles

NORD® Rare Disease Center of Excellence

Jennifer Cohen, MD

Assistant Professor of Pediatrics, Division of Medical Genetics

Duke University School of Medicine

NORD® Rare Disease Center of Excellence

John Concato, MD, MPH

Adjunct Professor

Yale School of Medicine

NORD® Rare Disease Center of Excellence

Christal Delagrammatikas, PhD

Director of Research

Malan Syndrome Foundation

Melissa Haendel, PhD, FACMI

Director, Precision Health & Translational Informatics; Sarah Graham Kenan Distinguished Professor

University of North Carolina at Chapel Hill

NORD® Rare Disease Center of Excellence

Collin Hovinga, PharmD, MS, FCCP

Vice President Rare Orphan and Pediatric Diseases

Critical Path Institute

Kerry Jo Lee, MD

Associate Director of Rare Diseases and Lead

Center for Drug Evaluation and Research (CDER), FDA

Mimi Lee, MD, PhD

Program Manager, Health Science Futures

Advanced Research Projects Agency for Health (ARPA-H)

Nara Lygia De Macena Sobreira, MD

Associate Professor of Genetic Medicine

Johns Hopkins University School of Medicine

NORD® Rare Disease Center of Excellence

Anabela Marçal, PharmD

EMA Liaison Official to US FDA

European Medicines Agency

Sydney Martinez, PhD, MPH

Associate Professor in Epidemiology

University of Oklahoma Health Sciences Center

Pramod Mistry, MD, PhD, FAASLD

Professor, Director, National Gaucher Disease Center

Yale University School of Medicine

NORD® Rare Disease Center of Excellence

Eva Morava-Kozicz, MD, PhD

Professor of Genetics and Genomic Sciences

Icahn School of Medicine at Mount Sinai

NORD® Rare Disease Center of Excellence

Patricia Musolino, MD, PhD

Associate Professor of Neurology, Harvard Medical School, Neurocritical Care and Vascular Neurologist

Massachusetts General Hospital / Harvard Medical School

NORD® Rare Disease Center of Excellence

Kiran Musunuru, PhD, MPH, ML, MRA

Professor of Cardiovascular Medicine, Genetics, and Pediatrics

Perelman School of Medicine at the University of Pennsylvania

NORD® Rare Disease Center of Excellence

Adora Ndu, PharmD, JD

Chief Regulatory Officer & EVP Portfolio Strategy and Management

BridgeBio

Scott Plotkin, MD, PhD

Giovanni ArmeniseEndowed Professor of Neurology, Harvard Medical School, Chief, Division of Neuro-Oncology

Mass General Brigham, Harvard Medical School

NORD® Rare Disease Center of Excellence

Vinay Prasad, MD, MPH

Director

Center for Biologics Evaluation and Research (CBER), FDA

Elizabeth Regan, MD, PhD

Research Professor of Medicine

National Jewish Health

Stephen Rosenfeld, MD, MBA

Executive Director

North Star Review Board

Rebecca Rothwell Chiu, PhD

Supervisory Mathematical Statistician

Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Lisa Forbes Satter, MD

Associate Professor of Pediatrics, Section of Immunology, Allergy and Rheumatology

Baylor College of Medicine and Texas Children’s Hospital

NORD® Rare Disease Center of Excellence

Maurizio Scarpa, MD, PhD

Director

European Reference Network for Hereditary Metabolic Diseases (MetabERN)

Sheri Schully, PhD

Deputy Chief Medical and Scientific Officer

NIH | All of Us Research Program

Fallon Schultz, MSW, LCSW, CAM

Co-Founder and CEO

International FPIES Association (IFPIES)

John Scott, PhD

Director, Division of Biostatistics

Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA)

Christopher Sibley, MD

Global Clinical Development Lead - Palopegteriparatide

Ascendis Pharma

Mark Skinner, JD

President and CEO

Institute for Policy Advancement Ltd.

Kevin S. Thorneloe, PhD

Senior Medical Director

Pharming Healthcare

Nicole Verdun, MD

Super Office Director, Office of Therapeutic Products

Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA)

Jerry Vockley, MD, PhD

Chief of Genetic and Genomic Medicine, Director of Center for Rare Disease Therapy

University of Pittsburgh Medical Center

NORD® Rare Disease Center of Excellence

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

Janet Woodcock, MD

Former Director

Center for Drug Evaluation and Research (CDER), FDA