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Thank you to our 2022 NORD Summit Speakers!

Vice President of Research
Institute for Clinical & Economic Review (ICER)

Foluso Agboola, MBBS, MPH

Deputy Director, Office of Clinical Evidence and Analysis, CDRH
US Food & Drug Administration (FDA)

Felipe Aguel, PhD

Executive Director
Hermansky-Pudlak Syndrome Network

Donna Appell, RN

Assistant Professor, Division of Medical Ethics
NYU Grossman School of Medicine

Alison Bateman-House, PhD, MPH

Senior Director, Federal Advocacy
American Academy of Pediatrics

James Baumberger, MPP

Executive Director, Head of Patient Advocacy and External Engagement
Pfizer, Inc

Katherine Beaverson, MS

Patient-Focused Drug Development Program Direction, CDER
US Food & Drug Administration (FDA)

Robyn Bent

RDCA-DAP Scientific Director
Critical Path Institute

Alex Betourne, PharmD, PhD

Executive Director of Strategic Partnerships
Amyloidosis Research Consortium (ARC)

Vanessa Boulanger, MSc

Program Director, US Regulatory Policy

Imein Bousnina, PharmD

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

PJ Brooks, PhD

Director of our Division of Services for Children with Special Healthcare Needs
US Department of Health and Human Services' Health Resources and Services Administration (HRSA)

Jeffrey Brosco, MD, PhD

Director, Office of Tissues and Advanced Therapies, CBER
US Food & Drug Administration (FDA)

Wilson Bryan, MD

Faculty, Bioethicist-Developmental Scientist
Boston Children's Hospital and Harvard Medical School

Lynn Bush, PhD, MS, MA

United States Commissioner of Food & Drugs

Robert Califf, MD

Senior Clinical Analyst for Stakeholder Engagements, CDER
US Food & Drug Administration (FDA)

Michelle Campbell, PhD

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

Patrizia Cavazzoni, MD

Rare Community Member
Peripartum Cardiomyopathy (PPCM) & Post-Transplant Lymphoproliferative Disorder (PTLD)

Brittany Clayborne, MS, PsyD

Rare Disease Policy Director
Little Hercules Foundation

Randi Clites

Associate Director for Real World Evidence Analytics, Office of Medical Policy, CDER
US Food & Drug Administration (FDA)

John Concato, MD, MS, MPH, BE

Executive Director
International Fibrodysplasia Ossificans Progressiva Association (IFOPA)

Michelle Davis

Director, Division of Rare Diseases and Medical Genetics, CDER
US Food & Drug Administration (FDA)

Kathleen Donohue, MD

Director of Membership
National Organization for Rare Disorders (NORD)

Debbie Drell

CEO & President
The E.WE Foundation

Sarita Edwards, BHSc

Associate Director, Oncology Center of Excellence; Deputy Director, Division of Oncology III- CDER/OND/OOD
Food and Drug Administration (FDA)

Lola Fashoyin-Aje, MD, MPH

Senior Managing Director
Manatt Health

Anthony Fiori

Director of Corporate Development
Cholangiocarcinoma Foundation

Lena Fung

Executive Director
Hemophilia Foundation of Southern California

Rigoberto Garcia, MPH

Global Head of Rare Disease Medical Affairs

Alaa Hamed, MD

Branch Chief, General Medicine 1, Office of Tissues and Advanced Therapies, CBER
US Food & Drug Administration (FDA)

Elizabeth Hart, MD

Program Director, Division of Clinical Research
National Institute of Neurological Disorders & Stroke, NIH

Adam Hartman

Senior Project Manager
Critical Path Institute

Laura Hopkins, MS

NORD Rare Cancer Coalition/Fibrolamellar Cancer Foundation

John Hopper


Mike Hu, PhD

Vice President of Medical and Scientific Strategy, Head of the Rare Disease Consortium
Syneos Health

Raymond Huml, MS, DVM, RAC

Rare Community Member & Founder
Living with Holt-Oram Syndrome

Nakisha Isom

Pediatric Team Leader, CDER
US Food & Drug Administration (FDA)

Mona Khurana, MD

Rare Community Member

Kayla Klein

Reviewer, Division of Clinical Outcome Assessment, CDER
US Food & Drug Administration (FDA)

Naomi Knoble, PhD

Professor of Pediatrics
Duke University School of Medicine

Dwight Koeberl, MD, PhD

Chief Medical Officer
Entrada Therapeutics

Nerissa Kreher, MD

President & CEO
Platelet Disorder Support Association (PDSA)

Caroline Kruse

Founder & Chief Executive Officer
Alliance to Cure Cavernous Malformation

Connie Lee, PsyD

Clinical Psychologist
Leidos Biomedical Research, Inc./National Cancer Institute

Robin Lockridge, PhD

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

Peter Marks, MD, PhD

Chief Executive Officer
Foundation for Sarcoidosis Research

Mary McGowan

Deputy Director of Science, Office of Pediatric Therapeutics, Office of the Commissioner
US Food & Drug Administration (FDA)

Ann McMahon

Head of US Regulatory Affairs, Global Rare Diseases
Chiesi USA Inc

Matt Medlin, PhD, RAC

Vice President Public Policy & Advocacy
Muscular Dystrophy Association

Paul Melmeyer

Program Director
National Institute of Neurological Disorders and Stroke, NIH

Jill Morris, PhD

Executive Director, Rare Disease COA Consortium
Critical Path Institute

Lindsey Murray, MPH, PhD

Assistant Professor of Clinical Medical and Molecular Genetics
Indiana University

Vinaya Murthy, MPH, MS LCGC

President & CEO
Indian Organization for Rare Diseases

Ramaiah Muthyala, PhD, MBA

Chief Regulatory Affairs Officer
BridgeBio Pharma

Adora Ndu, PharmD, JD

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

Edward Neilan, MD, PhD

Executive Director
TargetCancer Foundation

Jim Palma

Rare Community Member
MTHFS Gene Mutation

Annie Papik

Chief Medical Officer & Director for Pediatrics & Special Populations, CDRH
US Food & Drug Administration (FDA)

Vasum Peiris, MD

Vice President of Patient Services
National Organization for Rare Disorders (NORD)

Jill Pollander, RN, MSN

Medical Director, Children's National Rare Disease Institute
Children's National Hospital

Debra Regier, MD, PHD

Director of Community Outreach
Charcot-Marie-Tooth Association

Laurel Richardson

Associate Director of Pharmacoepidemiology
Oncology Center of Excellence, US Food & Drug Administration (FDA)

Donna Rivera, PharmD., MSc.

Director of Federal Policy
National Association of Medicaid Directors

Jack Rollins, MPH

Clinical Science Program Manager
Fanconi Anemia Research Fund

Andrea Ronan, MS, RD, CD, CNSC

President & CEO
TSC Alliance

Kari Rosbeck

Executive Director
National Ataxia Foundation

Andrew Rosen

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

Heidi Ross, MPH

Associate Director for Policy, Office of Tissues and Advanced Therapies, CBER
US Food & Drug Administratioin (FDA)

Anne Rowzee, PhD

President & Chief Executive Officer
National Organization for Rare Disorders (NORD)

Peter Saltonstall

President & Chief Executive Officer
Alpha-1 Foundation

Scott Santarella

Vice President of Community Engagement
National Organization for Rare Disorders (NORD)

Lisa Sarfaty

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

Jeffrey Shuren, MD, JD

Director, Adult Medical Genetics Program
University of Colorado, Anschutz Medical Campus

Matthew Taylor, MD, PhD

Director of Patient Education
Neuro Endocrine Tumor Research Foundation

Jessica Thomas, LCSW

Chief of Staff, Office of the Commissioner
US Food & Drug Administration (FDA)

Julie Tierney, JD

Branch Chief and Senior Investigator
National Heart, Lung, and Blood Institute, NIH

John Tisdale, MD

Senior Vice President and Head of Early Clinical
Denali Therapeutics

Matthew Troyer, MD

Chief of Genetic and Genomic Medicine, Director of the Center for Rare Disease Therapy
UPMC Childrens Hospital of Pittsburgh

Jerry Vockley, MD, PhD

United Porphyrias Association

Kristen Wheeden, MBA

Vice President, Director Patient Centered Research
Myositis Support & Understanding

Lynn Wilson

Chief Executive Officer
Youth Buddies

Kielan Wilson-Premo

Senior Medical Director, Rare Diseases

Ekaterina Wright

Senior Mathematical Statistician, CBER
US Food & Drug Administration (FDA)

Zhenzhen Xu

Office of Pediatric Therapeutics (PTC)
US Food & Drug Administration (FDA)

Sarah Zaidi, MD

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