Thank you to our 2020 speakers!

Associate Director of Advocacy
NORD

Kristen Angell

GFPD Diversity Committee Board Member, Sibling Caregiver and Advocate

Hannah Armstead

Director National Center for Advancing Translational Sciences
National Institutes of Health

Christopher Austin

Director
US Senate HELP Committee

Nick Bath


American Porphyria Foundation

Jennifer Beck

Professor, Division of Genetics and Metabolism Departments of Pediatrics and Genetics, Cell Biology & Development
University of Minnesota

Susan Berry

Associate Director for Patient Outcomes
Oncology Center of Excellence, FDA

Vishal Bhatnagar

Senior Vice President, Access and External Affairs
Genentech

Fritz Bittenbender

Director of Research Programs
NORD

Vanessa Boulanger

Associate Director of Research Programs
NORD

Danielle Boyce

Executive Director
Gorlin Syndrome Alliance

Julie Breneiser

VIce President, Global Commercial Development, Rare Disease
Pfizer Biopharmaceuticals Group

Bert Bruce

Co- Founder and Executive Director
The Global Foundation for Peroxisomal Disorders

Melissa Bryce Gamble

Associate Professor of Medicine, Vice Chair for Diversity and Inclusion Director, Nephrology Fellowship Program
Icahn School of Medice at Mount Sinai

Kirk Campbell

Deputy Director for Operations
Center for Drug Evaluation and Research(CDER) , Food and Drug Administration

Patrizia Cavazzoni

White House Presidential Innovation Fellow, Executive Office of the Presidents
Office of Science and Technology Policy

Dennis V. Chornenky

Director of Membership
NORD

Debbie Drell

CEO
Retrophin

Eric Dube

Deputy Director, Office of Medical Policy - Center for Drug Evaluation and Research
Food & Drug Administration

Khair El Zarrad

Co-founder and Executive Director of the Castleman Disease Collaborative Network (CDCN), Assistant Professor of Medicine in Translational Medicine & Human Genetics at the University of Pennsylvania, and Associate Director, Patient Impact for the Penn Orphan Disease Center

David Fajgenbaum

Chief Strategy Officer
NORD

Pamela Gavin

Professor of Pediatirics Medical Director, Kidney & Liver Tumor Programs Director, Advanced Cancer Therapeutics Network Director, Pilot Translational and Clinical Studies Program, CCTST
Cincinnati Children's Hospital Medical Center, Division of Oncology

James Gellar

Head, U.S. and Sanofi Genzyme Corporate Affairs
Sanofi Genzyme

Adam Gluck

President & Chief Executive Officer
Black Women's Health Imperative

Linda Gouler Blount

Managing Director
Sanofi Ventures

Jason Hafler

Commissioner
US Food and Drug Administration

Stephen Hahn

Associate Director, Quantitative Patient Inputs & RWE
Food and Drug Administration

Martin Ho


Critical Path Institiute, NORD Board Member

Kay Holcombe

Director, Center for Innovation in Global Health, Professor of Medicine, Georgetown University School of Medicine, and Distinguished Scholar and Program Director, O'Neill Institute for National and Global Health Law
Georgetown University

Charles Holmes

President
Fibrolamellar Cancer Foundation

John Hopper

Senior Director of Health Professional Programs
Aplastic Anemia & MDS International Foundation

Alice Houk

President & Founder
Foundation 29

Julian Isla

Acting Director
Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine at FDA

Hylton Joffe

General Partner
Sofinnova Investments

Maha Katabi

Senior Genomic Consultant
GeneDx

David Keane

Representative D- IL 2nd Disctrict
State of Illinois

Robin Kelley

Director
National Institute of Neurological Disorders and Stroke

Walter Koroshetz

Group Vice President, Oncology Targeted Therapeutics
Incyte

Peter Langmuir

Associate Director, DCEPT/OTAT Center for Biologics Evaluation and Research
Food and Drug Administration

Larissa Lapteva

Executive Director
Duchenne Regulatory Science Consortium, Rare Diseases Cures Accelerator- Data and Analytics Platform

Jane Larkindale

MSc, MCC, MMBA, Chief Patient Officer
Leo Pharma

Camilla Lauritzen

Chief Executive Officer
EURORDIS

Yann le Cam

Deputy Staff Director
House Committee on Energy & Commerce

Ryan Long

President & Chief Executive Officer
Global Blood Therapeutics

Ted Love

Co-Founder
Rallybio

Martin Mackay

Chief Executive Officer
Mito ACTION

Kira Mann

Director
CBER FDA

Peter Marks

State Representative, NH

Willam Marsh

Director, Office of Orphan Products Development
Food and Drug Administration

Janet Maynard

President and Chief Executive Officer
Biotechnology Innovation Organization (BIO)

Michelle McMurry-Heath

Board of Directors
CureSMA

Amy Medina

Founder, Chief Executive Officer
Hillstream BioParma

Randy Milby

Practice Director, Center for Healthcare Transformation
Avalere Health

Kristi Mitchell

Member, Board of Directors
NephCure Kidney Institute

Kevin Mott

Children's Hospital of Pittsburgh (CHOP)

Christine Munro

Chairman
Acceleron

Francois Nader

President & CEO
Alpha-1 Foundation

Miriam O’Day

Associate Director of Operations and Scientific Outreach for the Broad Cancer Program, Associate Director of Count Me In
Broad Institute

Corrie Painter

Executive Director
TargetCancer Foundation

Jim Palma

Founder & CEO
The Mighty

Mike Porath

Management Analyst, Office of Rare Diseases Research (ORDR)
National Center for Advancing Translational Sciences (NCATS)

Deanna Portero

Health Policy Advisor
US Senate Committee on Finance

Stuart Portman

Senior Policy Advisor on Budget and Health to Congresswoman Nancy Pelosi (D-CA)

Wendell Primus


Childrens National and the Rare Disease Institute

Debra Regier

Director
Rate Tumor Initiative, CCR, NCI

Karlyne Reilly


Food & Drug Administration

Donna Roscoe

Director Of Policy
NORD

Heidi Ross

President and CEO
NORD

Peter Saltonstall

Vice President for Research and Chief Scientific Officer
CHOC Children's Hospital of Orange County

Terence Sanger

Director of Strategic Planning & Project Development
NORD

Lisa Sarfaty


Scheer & Company Inc

David Scheer

Senior Research Programs Manager
NORD

Allison Seebald

President/Executive Director
ADNP Kids Research Foundation

Sandra Sermone

Founder & CEO
Applied Therapeutics

Shoshana Shendelman

Sr. Scientific Director, Global Medical Affairs
Novartis Gene Therapies

Lydia Shenouda

Vice President, Policy and Regulatory Affairs
NORD

Rachel Sher


Eversana

Oodaye Shukla


Cystic Fibrosis Foundation

Mark Sleeper

Global Medical Lead, Hemophilia
BioMarin Pharmaceutical Inc

Paul Solari

Associate Professor in Psychiatry, Neurology and Pediatrics NORD Board Member
UCLA David Geffen School of Medicine Los Angeles, CA

Shafali Spurling Jeste

Health Science Policy Analyst
Food & Drug Administration

Heather Stone

Director of ResearchPrograms
Foundation for Prader-Willi Research

Theresa Strong

Professor of Pediatrics, Wallace Chair of Pediatrics Division of Allergy Immunology
CHOP (Children's Hopsital of Philadelphia)

Kathleen Sullivan

Division Chief, Genetics and Metabolism, Director, Rare Disease Institute
Children's National

Marhsall Summar

Wisconsin Rare Action Network Volunteer State Ambassador
NORD

Danyelle Sun

Assistant Professor, Director VIDE Program Horae Gene Therapy Center, Department of Microbiology and Physiological Systems
University of Massachusetts Medical School

Phillip Tai


Rare Action Network, NORD

Brooke Thomas

Chief Executive Officer
Board Veritas

Lisa Anne Thompson Taylor

Director of Research
Association for Creatine Deficiencies

Laura Trutoiu

Director, Clinical Trial Services Director, Ocular & Rare Disease Genetics Services
InfomedDNA

Karmen Trzupek

Head, Organic Acid Research Section Senior Investigator, National Human Genome Research Institute
National Institutes of Health

Charles Venditti

Executive Director
Dup15q Alliance

Vanessa Vogel-Farley

Senior Director Clinical Development
Foundation Medicine Inc.

Ashley Ward

Deputy Director National Institute on Minority Healh and Health Disparities
National Institutes of Health

Monica Webb Hooper

President and CEO
Canadian Organization for Rare Disorders

Durhane Wong-Rieger

Executive Director
International Pemphigus and Pemphigoid Foundation (IPPF)

Mark Yale

VP of Biological Development
Editas Medicine

Kate Zhang

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