Speakers

Michael Bagel, Esq.

Vice President, Policy

Alliance of Community Health Plans

Jay Bhattacharya, MD, PhD

Director

National Institutes of Health (NIH)

Amy Comstock Rick, JD

Associate Director for Rare Disease Strategy; Director of Strategic Coalition

CDER; FDA Rare Disease Innovation Hub

Elizabeth Ames, MD, PhD

Clinical Associate Professor of Pediatrics

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

Lisa Butler

President & CEO

GBS | CIDP Foundation International

Daniel Caños, PhD

Director, Office of Clinical Evidence and Analysis, OPEQ

CDRH, FDA

Urania Dagalakis, MD, MHSc

Co-Director, Cancer Genetics Clinic; Assistant Professor of Pediatrics

Children’s National Hospital, a NORD Rare Disease Center of Excellence; George Washington University

Michael Davis, MD, PhD

Director

CDER, FDA

Aneesh Deoras, MSE

Acting Division Director for Digital Health Technology Assessment

CDRH, FDA

Kyle Diamantas, JD

Acting Commissioner of Food and Drugs

U.S. Food and Drug Administration (FDA)

Danielle Dong

Scientific Advocacy Lead, Global Medical Affairs, Rare Disease

Sanofi

Sarah Dunsmore, PhD

Program Director

NCATS, NIH

Stephanie E. Haridopolos, MD, DABFM

Director of National Health Communications

Office of the Surgeon General and Principal Deputy Assistant Secretary for Health Policy

Renee Gala

President and CEO

Jazz Pharmaceuticals

Pamela Gavin, MBA

President and Chief Executive Officer

NORD

Hilary Gee Goeckner, MSW

Director, State & Local Campaigns - Access to Care

American Cancer Society Cancer Action Network

Steven Gray, PhD

Professor

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

Cynthia Grossman, PhD

Director, Division of Patient-Centered Development

OID, OST, CDRH, FDA

Ada Hamosh, MD, MPH

Dr. Frank V. Sutland Professor of Pediatric Genetics, Department of Genetic Medicine and Pediatrics

Johns Hopkins Medicine, a NORD Rare Disease Center of Excellence

Alexander Hardy

President and CEO

BioMarin

Tracy Hart

CEO

Osteogenesis Imperfecta Foundation

Elizabeth Hart, MD

Associate Director for Rare Diseases

CDER, FDA

Regina Hartfield

President and CEO

Sickle Cell Disease Association of America, Inc.

TaLana Hughes, MPH

Executive Director

Sickle Cell Disease Association of Illinois

Reena Kartha, MS, PhD

Associate Professor & Senior Associate Director

University of Minnesota Center for Orphan Drug Research, a NORD Rare Disease Center of Excellence

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

Eyby Leon, MD

Director of the Medical Genetics Residency Program

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

Kathryn Lowell, MPA

Executive Vice President, Government Affairs

NORD

Martin Mackay, PhD

Co-Founder and Chairman of the Board

RallyBio

Marybeth McAfee, MA, GC

Vice President, Community and Medical Affairs

NORD

Karim Mikhail, B.Pharm, MSc

Director

CBER, FDA

Nicholas Minter

Director, Seamless Care Models Group

CMS Innovation Center

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

Director, Neuromuscular Center

Cleveland Clinic, a NORD Rare Disease Center of Excellence

Sean Nolan

Chief Executive Officer

Taysha Gene Therapies

Jim Palma

CEO

TargetCancer Foundation

Becky Quick

Co-anchor and Host

CNBC “Squawk Box” and CNBC Cures

Tarek Rabah

President and Chief Executive Officer

Otsuka North America Pharmaceutical

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

Heather Saunders, PhD

Senior Research Manager

KFF

David Scheer

President

Scheer & Company, Inc.

Brittany Schuck, PhD

Lead, CDRH Rare Disease Impact Initiative, Deputy Office Director

OHT7: Office of In Vitro Diagnostics, CDRH, FDA

Ben Shaberman, MS, MA

Science Communications Advisor

Hope in Focus

Carolyn G. Sheridan, MPH

Associate Director of State Policy

NORD

Tracey Sikora

Vice President of Research and Clinical Programs

NORD

The Honorable Troy Singleton, BS

New Jersey State Senator

New Jersey Legislature

Stephen Squinto, PhD

Chief Investment Officer

J.P. Morgan Life Science Private Capital

Theresa Strong, PhD

Director of Research Programs

Foundation for Prader-Willi Research

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

Cara Tenenbaum, JD, MBA

Director of Regulatory Affairs

NORD

Nicholas Vita

Co-Founder, CEO, Member of the Board

Apriligen, Inc.

Janet Woodcock, MD

Former Director

Center for Drug Evaluation and Research, FDA

Naveena Yanamala, MD, PhD, FASE

Section Chief of Clinical Research and AI Innovation and Director of Data Science and Machine Learning

Rutgers, Robert Wood Johnson, a NORD Rare Disease Center of Excellence

Byeong Yoon

Vice President of Medical, Rare Disease

Amgen