Now Accepting Speaker Applications for the 2024 NORD Breakthrough Summit!

The NORD Breakthrough Summit will be held October 20-22, 2024

NORD is seeking expert speakers to present on the following topics:

  • Innovation in Accelerating Diagnosis
  • Closing the Gap Between Diagnosis and Clinical Care
  • New Approaches to Patient Centric Clinical Trials
  • The Use of Data in Effective Patient Focused Drug Development
  • The Role of Advocacy in Driving Change
  • Unique Programming at Patient Advocacy Organizations
  • Patient Voice: Engagement and Experience in the Rare Disease Space
  • Health Equity in Rare Diseases
  • Pathways to Patient Access: Drugs Pricing, Insurance, Healthcare and More
  • And Many Others!

Speaker Proposal Guidelines 

As you prepare your submission, please take note of the following:   

  • Proposals should be geared towards all members of the NORD Breakthrough Summit audience, which includes leaders from industry, government, academia, research and patient advocacy organizations.   
  • Talks solely intended for patients and caregivers should be submitted via the speaker application process for the Living Rare, Living Stronger NORD Patient and Family Forum. 
  • Due to the diverse audience, academic disease-specific/scientific talks will not be accepted.  
  • NORD seeks proposals that are disease agnostic or of which learnings could be applicable for more than one disease state.  
  • Inclusion of video/audio samples will be prioritized in speaker selection.  
  • Please note that NORD most often invites speakers to participate in a moderated panel discussion to allow for a conversational session with different perspectives included. If you are invited to speak, though NORD may select your topic, it may be used for a panel discussion rather than a podium presentation as submitted in this application. 

All speaker submissions must be received through the NORD website no later than Friday, March 8, 2024 at 11:59pm ET. 

2023 NORD Breakthrough Summit Speakers

Deputy Director Division of Pediatric & Maternal Health, Office of New Drugs, CDER
US Food & Drug Administration (FDA)

John Alexander, MD, MPH

Rare Community Member

Rixys Alfonso, Patient

Rare Community Member

Devin Alvarez, Patient

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

Office of Strategic Partnerships & Technology Innovation, CDRH
US Food & Drug Administration (FDA)

Fraser Bocell, MED, PhD

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

Chief of Pediatrics
Boston Children's Hospital

Wendy Chung, MD, PhD

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


Alex Flipse, Patient

Freedman Counseling Associates

Albert Freedman, PhD

Office of Neuroscience, Office of New Drugs, CDER

Emily Freilich, MD

Founder and Chief Innovation Officer
Deep Genomics

Brendan Frey, 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

Director, Office of Pediatric Therapeutics, OCPP, OC

Dionna Green, MD

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

Chief Technology Officer
GE Healthcare

Taha Kass-Hout, MD, MS

SVP, Chief Medical Officer
Reata Pharmaceuticals

Seemi Khan, MD, MPH, MBA

Senior Vice President for Healthcare Policy & Research
Biotechnology Innovation Organization (BIO)

Crystal Kuntz

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

Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine; OND, CDER

Janet Maynard, MD

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

President & Chief Operating Officer
Rocket Pharmaceuticals

Kinnari Patel, PharmD, MBA

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
OrphAI Therapeutics

Brigette Roberts, MD


Luke Rosen, MSc

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

Heidi Ross, MPH

Global Chairman, Healthcare Investment Banking
JP Morgan

Philip Ross, MD

Vice President, External Affairs

Joy Russell

Scheer & Company

David Scheer

Senior Health Policy Advisor
Senate Committee on Finance

Conor Sheehey

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

Office of Medical Policy, CBER
Food & Drug Administration

Heather Stone, MPH

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

Michelle Tarver, MD, PhD

Co-Founder and Chief Executive Officer

Stephen Uden, MD

Senior Director (Digital Health & Technology)
Medical Device Innovation Consortium (MDIC)

Jitesh Veetil, PhD

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

Mark Veich

Director, Office of Therapeutic Products, CBER

Nicole Verdun, MD

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

Division of Rare Diseases & Medical Genetics, Deputy Director, Office of New Drugs, CDER
US Food & Drug Administration (FDA)

Yuliya Yasinskaya, MD

Venture Partner

Tal Zaks, MD, PhD

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