2019 Confirmed Speakers

Conference Keynote Speakers

David Alexanderian

Global Clinical Development Le...

Takeda Pharmaceutical

Alix Alderman

VP Head of Regulatory Affairs ...

PellePharm

Daniel Anderson

Head Commercial Partnerships

Invitae

Sonali Chopra

Director Alliance & Advocacy R...

Genentech

damikolas

Allyson Damikolas

Mother & Carer

Rare Disease Patient

Khrystal K. Davis

CEO and Patient Advocate

Zebra Leaf Publishing

Rachelle Dixon

President / Co-Founder

HSAN1E Society

Carl K Edwards

Chief Scientific Officer

DNX Biopharmaceuticals

Clasina Field

Co-Executive Director

Bens Friends

Jacob Fraker

Legislative Associate

Cystic Fibrosis Research, Inc. (CFRI)

Bruno Gagnon

Senior Vice President, Develop...

Eidos Therapeutics Inc.

Todd Galles

Business Development Principal

Durbin

Femida Gwadry‐Sridhar

Founder and CEO

Pulse Infoframe Inc.

Fred Holdbrook

Director of Biostatistics

Amicus Therapeutics

T Anthony Howell

Co-Founder & Digital Community...

rareLife solutions

Lucas Kempf

Medical Officer for the Rare D...

FDA

Tom Lambdin

Co-Executive Director

Ben’s Friends

David Y. Liu

Chief Scientific Officer, Head...

Protagonist Inc

Qing Liu

Founder & Principal Consultant

QRMedSci

Serge Messerlian

President

Actelion Pharmaceuticals US, Inc

Debra Miller

CEO

CureDuchenne

Eric M. Mueller

Associate Director, Cell Thera...

Orchard Therapeutics

Angela Ramirez Holmes

Founder/President

Cal Rare

Harsha Karur Rajasimha

Co-Founder, Board Director

George Mason University, Fairfax, VA

Javier San Martin

Vice President Clinical Develo...

Ultragenyx

Vij Senthilnathan

Senior Clinical Study Manager

BioElectron Technology Corporation

James P. Shaffer

MBA, Chief Business Officer

Eiger BioPharmaceuticals

Mridula Shukla

Global Program Lead, Regulator...

Adverum Biotechnologies, Inc.

Katherine Stueland

Chief Commercial Officer

Invitae

Michael A. Swit

FDA Legal Counsel

Law Offices of Michael A. Swit

Christine Von Raesfeld

Patient Advocate, More Than Lu...

PatientsLikeMe

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