Speakers

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

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

Melissa Haendel, PhD, FACMI

Department of Genetics

University of North Carolina at Chapel Hill; Monarch Initiative

NORD® Rare Disease Center of Excellence

Collin Hovinga, PharmD, MS, FCCP

Vice President of the Rare and Orphan Disease Programs

Critical Path Institute, Rare Disease Cures Accelerator – Data Analytics Platform (RDCA-DAP ® )

Nara Lygia De Macena Sobreira, MD

Associate Professor of Genetic Medicine

Johns Hopkins School of Medicine

NORD® Rare Disease Center of Excellence

Anabela Marçal, PharmD

EMA Liaison Official to US FDA

European Medicines Agency

Pramod Mistry, MBBS, PhD, MA, MD

Professor of Medicine (Digestive Diseases) and of Pediatrics (Gastroenterology)

Yale 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

Pediatric Neurology

Massachusetts General Hospital, Harvard Medical School

NORD® Rare Disease Center of Excellence

Kiran Musunuru, PhD, MPH, ML

Director of the Genetic and Epigenetic Origins of Disease Program

University of Pennsylvania

NORD® Rare Disease Center of Excellence

Adora Ndu, PharmD, JD

Chief Regulatory Officer and Executive Vice President-Portfolio Strategies 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

Stephen Rosenfeld, MD, MBA

Executive Director

North Star Review Board

Lisa Forbes Satter, MD

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

Baylor College of Medicine and Texas Children’s Hospital

NORD® Rare Disease Center of Excellence

Fallon Schultz, MSW, LCSW

Co-Founder and CEO

International FPIES Association (IFPIES)

Mark Skinner, JD

President and CEO

Institute for Policy Advancement Ltd.

Kevin S. Thorneloe, PhD

Senior Medical Director

Pharming Healthcare

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

Lurie Children’s Hospital of Chicago

NORD® Rare Disease Center of Excellence

Janet Woodcock, MD

Former Director

Center for Drug Evaluation and Research, FDA