Speakers

Jay Bhattacharya, MD, PhD

Director

National Institutes of Health (NIH)

Elizabeth Ames, MD, PhD

Clinical Associate Professor of Pediatrics

University of Michigan Health, a NORD Rare Disease Center of Excellence

Kyle Diamantas, JD

Acting Commissioner of Food and Drugs

U.S. Food and Drug Administration (FDA)

Renee Gala

President and Chief Executive Officer

Jazz Pharmaceuticals

Pamela Gavin

Chief Executive Officer

NORD

Steven Gray, PhD

Professor

UT Southwestern Medical Center, a NORD Rare Disease Center of Excellence

Alexander Hardy

President and Chief Executive Officer

BioMarin Pharmaceutical

Tracy Hart

CEO

Osteogenesis Imperfecta Foundation

Elizabeth Hart, MD

Associate Director for Rare Diseases

CDER, FDA

Reena Kartha, MS, PhD

Associate Professor & Associate Director

Center for Orphan Drug Research, Experimental and Clinical Pharmacology, University of Minnesota

Beth Kozel, MD, PhD

Chief of the Division of Genetics and Genomic Medicine and the Director of Constitutional Genomics Translational Research

Nationwide Children's Hospital, a NORD Rare Disease Center of Excellence

Martin Mackay, PhD

Co-Founder & Chair

RallyBio

Marybeth McAfee, MA, GC

Vice President, Community and Medical Affairs

NORD

John A. Morren, MD, MEd, FAAN, FAANEM

Director

Cleveland Clinic Lerner College of Medicine of Case Western Reserve University

Sean Nolan

Chief Executive Officer

Taysha Gene Therapies

Tarek Rabah, MBA

President and Chief Executive Officer

Otsuka North America Pharmaceutical Business

Debra Regier, MD, PhD

Director

Ward Winslett Center for Rare Diseases, Atrium Health Levine Children's Hospital, a NORD Rare Disease Center of Excellence

Lindsay Rosen

Vice President, Strategy

Medlive

Camille Samuels

Board Director and Venture Investor

David Scheer

President

Scheer & Company, Inc.

Ben Shaberman, MS, MA

Science Communications Advisor

Hope in Focus

Natasha Shur, MD

Division Chief, Genetics and Metabolism, Medical Geneticist

Children’s National Hospital

Stephen Squinto, PhD

Chief Investment Officer

J.P. Morgan Life Science Private Capital

Uthra Sundaram

Senior Vice President, Business Unit Head

USBU Foundational Therapeutics Platform (FTP); Takeda Pharmaceuticals U.S.A., Inc.

V. Reid Sutton, MD

Professor of Molecular & Human Genetics

Baylor College of Medicine & Texas Children’s Hospital, a NORD Rare Disease Center of Excellence

Michelle E. Tarver, MD, PhD

Director

Center for Devices and Radiological Health (CDRH), FDA

Janet Woodcock, MD

Former Director

Center for Drug Evaluation and Research, FDA

Byeong Yoon

Vice President of Medical, Rare Disease

Amgen