2023 NORD Breakthrough Summit Speakers

Rare Community Member

Rixys Alfonso

Rare Community Member

Devin Alvarez

CSO and Head of Rare Diseases
Acadia Pharmaceuticals

Kathie Bishop, PhD

Senior Program Manager, Science in Society, and Lead, Rare As One Network
Chan Zuckerberg Initiative

Heidi Bjornson-Pennell

Chief Science Officer
Kabuki Syndrome Foundation

Bruce Bloom, JD, DDS

Associate Chief of Genetics & Genomics
Boston Children's Hospital

Olaf Bodamer, MD, PhD

Acting Director, Division of Rare Diseases Research Innovation, National Center for Advnacing Translational Sciences (NCATS)

PJ Brooks, PhD

Chief Commercial Officer

Jennifer Butler

US Food & Drug Administration (FDA)

Robert Califf, MD

Founder and Director
Neurology Rare Disease Center

Diana Castro, MD

Director, Center for Drug Evaluation & Research (CDER)
US Food & Drug Administration (FDA)

Patrizia Cavazzoni, MD

Geneticist & Metabolist
Children's National Hospital

Kimberly Chapman, MD, PhD

Senior Vice President, Research & Product Development
Alexion/AstraZeneca Rare Disease

Seng Cheng, PhD

Executive Director, Head of Global R&D and Regulatory Policy
BioMarin Pharmaceutical Inc.

Nimi Chhina, PhD, JD

Vice President, Government Relations
Alliance for Regenerative Medicine

Erica Cischke, MPH

General Manager, US Rare Diseases

R. Duane Clark

VP of Corporate Relations
National Organization for Rare Disorders (NORD)

Patrick Collins

Director, Patient Advocacy
Horizon Therapeutics

Tom D'Amato

President & Chief Executive Officer
Travere Therapeuticcs

Eric Dube, PhD

Chief Scientific Officer
The FamilieSCN2A Foundation

Shawn Egan, PhD

Director of Research Data and Analytics
National Organization for Rare Disorders (NORD)

Aliza Fink, DSc

Freedman Counseling Associates

Albert Freedman, PhD

Chief Medical Officer
Children's Tumor Foundation

Brigid Garelik, MD MPH

Rogers Behavioral Health

Sheldon Garrison, PhD

Medical Director
Progeria Research Foundation

Leslie Gordon, MD, PhD

Vice President, Head, GRA Global Product Strategy
CSL Behring

Scott Hambaugh, MBA

Director, Policy and Regulatory Affairs
National Organization for Rare Disorders (NORD)

Karin Hoelzer, DVM, PhD

Vice President of the Rare and Orphan Disease Programs
Critial Path Institute

Collin Hovinga, PharmD, MS, FCCP

ATI Advisory

Anna Kaltenboeck

SVP, Chief Medical Officer
Reata Pharmaceuticals

Seemi Khan, MD, MPH, MBA

Investigator, Professor
Broad Institute; Harvard University

David Liu, PhD

Professor of Pediatrics & Chief Division of Medical Genetics
University of Utah, Spencer Fox Eccles School of Medicine

Nicola Longo, MD, PhD

Clinical Health Psychologist, Professor of Pediatrics
Children's National Hospital

Maureen Lyon, PhD

Co-Founder and Executive Chairman

Martin Mackay, PhD

Director, Center for Biologics Evaluation and Research (CBER)
US Food & Drug Administration (FDA)

Peter Marks, MD, PhD

Chief of Staff, Center for Medicare
Center for Medicare and Medicaid Services (CMS)

Kristi Martin

Vice President, Decentralized Clinical Trials (DCT)

Kelly McKee

To Cure A Rose Foundation

Casey McPherson

Chief Medical Officer
Orchard Therapeutics

Leslie Meltzer, PhD

CEO & Founder
Folia Health

Nell Meosky Luo

Head of US Rare Diseases

Kimberly Moran, PhD

Director, Orphan Products Grants Program, OOPD
US Food & Drug Administration (FDA)

Katherine Needleman, MS, PhD, RAC

Chief Medical & Scientific Officer
National Organization for Rare Disorders (NORD)

Edward Neilan, MD, PhD

Chief Global Development Officer
The Marfan Foundation

Eileen Novins-Masciale, MA

Rare Disorder Nurse Navigator
Washington University School of Medicine, St. Louis

Michelle Oliva, MSN, RN

Head of Health Equity Alliances & Partnerships, Center for Health Equity & Patient Affairs

Linn Parrish

Chief Medical Officer and Director, Pediatrics & Special Populations, Centers for Devices and Radiological Health
US Food & Drug Administration (FDA)

Vasum Peiris, MD, MPH

President & Chief Executive Officer
Sumitomo Pharma America

Myrtle Potter

Director, Office of Orphan Products Development (OOPD)
US Food & Drug Administration (FDA)

Sandra Retzsky

Chief Executive Officer
AI Therapeutics

Brigette Roberts, MD


Luke Rosen, MSc

Global Chairman, Healthcare Investment Banking
JP Morgan

Philip Ross, MD

Vice President, Policy & Regulatory Affairs
National Organization for Rare Disorders (NORD)

Heidi Ross, MPH

Vice President, External Affairs

Joy Russell

Scheer & Company

David Scheer

Senior Health Policy Advisor
Senate Committee on Finance

Conor Sheehey

Chief Advocacy Officer
Biotechnology Innovation Organization (BIO)

Nick Shipley

Director, Center for Devices & Radiological Health
US Food & Drug Administration (FDA)

Jeff Shuren, MD, JD

Program Officer
NIH, NCATS, Division of Rare Diseases Research Innovation

Eric Sid, MD, MHA


Tracey Sikora

Chief Medical Officer
Beam Therapeutics

Amy Simon, MD

Associate Professor
Johns Hopkins University

Nara Sobreira, MD, PhD

Director, Office of Strategy & Alliances Pediatric Translational Neuroscience Initiative
St. Jude Children's Research Hospital

Kristin Stephenson, MHA, JD

Deputy Center Director for Transformation, CDRH
US Food & Drug Administration (FDA)

Michelle Tarver, MD, PhD

Co-Founder and Chief Executive Officer

Stephen Uden, MD

Vice President, Philanthropy, and Executive Director of the Deerfield Foundation

Mark Veich

United Porphyrias Association

Kristen Wheeden

Deputy Director, Center for Biologics Evaluation & Research (CBER)
US Food & Drug Administration (FDA)

Celia Witten, MD, PhD

Office of Clinical Evaluation, Office of Therapeutic Products , CBER
US Food & Drug Administration (FDA)

Lei Xu, MD, PhD

Venture Partner

Tal Zaks, MD, PhD

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