NORD Breakthrough Summit 2022 Speakers

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

RDCA-DAP Scientific Director
Critical Path Institute

Alex Betourne

Executive Director of Strategic Partnerships
Amyloidosis Research Consortium (ARC)

Vanessa Boulanger, MSc

Program Director, US Regulatory Policy

Imein Bousnina, Pharm.D

Acting Director, Division of Rare Diseases Research Innovation

PJ Brooks, 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

Lynn Bush, PhD, MS, MA

Center for Drug Evaluation & Research (FDA)

Patrizia Cavazzoni, MD

Rare Community Member
Lymphoproliferative Disorder (PTLD)

Brittany Claybourne, MS, PsyD

Executive Director
International Fibrodysplasia Ossificans Progressiva Association (IFOPA)

Michelle Davis

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

Kathleen Donohue, MD

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

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

Elizabeth Hart, MD

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

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

Naomi Knoble, PhD

Founder & Chief Executive Officer
Alliance to Cure Cavernous Malformation

Connie Lee, Psy.D

Clinical Psychologist
National Cancer Institute

Robin Lockridge, PhD

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

Peter Marks, MD, PhD

Program Director
National Institute of Neurological Disorders and Stroke

Jill Morris, PhD

Executive Director, Rare Disease COA Consortium
Critical Path Institute

Lindsey Murray

Assistant Professor of Clinical Medical and Molecular Genetics
Indiana University

Vinaya Murthy, MPH, MS LCGC

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

President & CEO
TSC Alliance

Kari Rosbeck

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

Senior Research Programs Manager
National Organization for Rare Disorders (NORD)

Allison Seebald

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

Senior Investigator and Director
National Center for Advancing Translational Science (NCATS), NIH

John Tisdale

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

Jerry Vockley, MD, PhD

United Porphyrias Association

Kristen Wheeden, MBA

Chief Executive Officer
Youth Buddies

Kielan Wilson-Premo

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

Zhenzhen Xu

Keep up-to-date on the latest NORD Summit news