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Speakers

Explore Patient-Centric Approaches to Trial Design, Recruitment and Access to Enhanced Outcomes

December 3-4, 2019
  • Arlington, VA

Melanie Blank, MD
Medical Officer, Rare Diseases Program
U.S. Food and Drug Administration (FDA)

Ron Cooper
President & CEO
Albireo Pharma

Michael Eging
Executive Director
Rare Action Access Project

Chris Garabedian
CEO
Xontogeny

Derrick Gingery
Senior Writer
The Pink Sheet and Scrip

Nielsen Hobbs
Editor
The Pink Sheet

Emil D. Kakkis, MD, PhD
CEO, President and Founder
Ultragenyx Pharmaceutical Inc.

Jaswinder Singh Khera
Managing Director Americas
WEP Clinical

Brett Kopelan
Executive Director
debra of America

Nanxin (Nick) Li, PhD, MBA
Senior Director, Health Economics and Outcomes Research
uniQure

Candace Lerman
Founder
Rare Candace

Peter Marks, MD, PhD
Director, Center for Biologics Evaluation and Research (CBER)
U.S. Food and Drug Administration (FDA)

John Maslowski
CEO
Fibrocell

Ali Mohamadi
Executive Director, Patient Advocacy
BioMarin Pharmaceutical

Sara Nayeem, MD
Partner
NEA

Adora Ndu, PharmD, JD
Executive Director, Regulatory Affairs, Policy,
Research, Engagement (PRE), & Reg Internationa
BioMarin Pharmaceutical

Nneka Onwudiwe, PharmD, PhD, MBA
Founder and Chief Executive Officer
Pharmaceutical Economics Consultants of America (PECA) LLC

Tejashri Purohit-Sheth, MD
Director, Division of Clinical Evaluation and
Pharmacology/Toxicology (CBER / OTAT / DCEPT)
U.S. Food and Drug Administration (FDA)

Kari Rosbeck
President & CEO
Tuberous Sclerosis Alliance

Len Rosenberg
Head of Clinical Operations
Beat AML & Leukemia and Lymphoma Society

Christian Rubio
Vice President Community Development & Engagement
Global Genes

Lyndsey Scull
Vice President Communications
Alliance for Regenerative Medicine

Peter Stein
Director, Office of New Drugs, CDER
U.S. Food and Drug Administration (FDA)

Dan Takefman, Ph.D.
Head, Regulatory Affairs
Spark Therapeutics

Monica Weldon
President, CEO & Founder
Bridge the Gap – SYNGAP Education and Research Foundation

Michael Werner
Co-Founder and Senior Policy Counsel
Alliance for Regenerative Medicine

Melanie Blank, MD
Medical Officer, Rare Diseases Program
U.S. Food and Drug Administration (FDA)

Ron Cooper
President & CEO
Albireo Pharma

Michael Eging
Executive Director
Rare Action Access Project

Chris Garabedian
CEO
Xontogeny

Derrick Gingery
Senior Writer
The Pink Sheet and Scrip

Nielsen Hobbs
Editor
The Pink Sheet

Emil D. Kakkis, MD, PhD
CEO, President and Founder
Ultragenyx Pharmaceutical Inc.

Jaswinder Singh Khera
Managing Director Americas
WEP Clinical

Brett Kopelan
Executive Director
debra of America

Nanxin (Nick) Li, PhD, MBA
Senior Director, Health Economics and Outcomes Research
uniQure

Candace Lerman
Founder
Rare Candace

Peter Marks, MD, PhD
Director, Center for Biologics Evaluation and Research (CBER)
U.S. Food and Drug Administration (FDA)

John Maslowski
CEO
Fibrocell

Ali Mohamadi
Executive Director, Patient Advocacy
BioMarin Pharmaceutical

Sara Nayeem, MD
Partner
NEA

Adora Ndu, PharmD, JD
Executive Director, Regulatory Affairs, Policy,
Research, Engagement (PRE), & Reg Internationa
BioMarin Pharmaceutical

Nneka Onwudiwe, PharmD, PhD, MBA
Founder and Chief Executive Officer
Pharmaceutical Economics Consultants of America (PECA) LLC

Tejashri Purohit-Sheth, MD
Director, Division of Clinical Evaluation and
Pharmacology/Toxicology (CBER / OTAT / DCEPT)
U.S. Food and Drug Administration (FDA)

Kari Rosbeck
President & CEO
Tuberous Sclerosis Alliance

Len Rosenberg
Head of Clinical Operations
Beat AML & Leukemia and Lymphoma Society

Christian Rubio
Vice President Community Development & Engagement
Global Genes

Lyndsey Scull
Vice President Communications
Alliance for Regenerative Medicine

Peter Stein
Director, Office of New Drugs, CDER
U.S. Food and Drug Administration (FDA)

Dan Takefman, Ph.D.
Head, Regulatory Affairs
Spark Therapeutics

Monica Weldon
President, CEO & Founder
Bridge the Gap – SYNGAP Education and Research Foundation

Michael Werner
Co-Founder and Senior Policy Counsel
Alliance for Regenerative Medicine