Speakers

Vanessa Acero

LPCC-Licensed Mental Health Therapist

Miguel Fernandez Alcalde

President

EMD Serono

Danaé Bartke

Executive Director

HCU Network America

Claire Beggs

Executive Director, Global Regulatory Affairs and Drug Safety

Jazz Pharmaceuticals

Bruce Bloom

Chief Science Officer

Kabuki Syndrome Foundation

R. Duane Clark

General Manager, U.S. Rare Diseases

Sanofi

Patrick Collins

Vice President of Community & Corporate Affairs

NORD

Terence Flotte, MD

President

American Society for Gene & Cell Therapy (ASGCT)

Stephanie Fradette, MD

Head of Neuromuscular Development Unit

Biogen

Amy Gaviglio, MS, CGC

Founder and Chief Executive Officer

Connetics Consulting LLC

Pamela Gavin

Chief Executive Officer

NORD

Sarah Gheuens, MD, PhD

Chief Medical Officer, Head of R&D

Agios

Kaushik Ghosal, PhD

Amyloidosis Research Consortium ASPIRE Collaboration

John Greally, MD

Children’s Hospital at Montefiore Einstein

Allison Herrity, MPH

Senior Policy Analyst

NORD

Nerissa Kreher, MD, MBA

Chief Medical Officer

Alltrna

Martin Mackay, PhD

Co-Founder

RallyBio

Ray Merenstein

Parent, Director of NAMI CO

Colorado Rare Disease Advisory Council

Vinod Mitta

Director

Seamless Care Models Group, CMS Innovation Center, US HHS

Edward Neilan, MD, PhD

Chief Medical and Scientific Officer

NORD

Sean Nolan

Chief Executive Officer

Taysha Gene Therapies

Catherine Owen Adams, BSc.

Chief Executive Officer

Acadia Pharmaceuticals

Jill Pollander, RN, MSN

Vice President of Patient Services

NORD

Mike Porath

Chief Executive Officer and Founder; Executive Director

The Mighty; Dup15q Alliance

Maha Radhakrishnan, MD

Executive Partner

Sofinnova Investments

Paula Ragan

Chief Executive Officer and President

X4 Pharmaceuticals

Amy Rick, JD

Director of Strategic Coalitions

FDA’s Rare Disease Innovation Hub

Dylan Ritter, PhD

Director of Scientific and Clinical Initiatives

Dup15Q Alliance

Philip Ross

Vice Chairman

Jefferies LLC

David Scheer

President

Scheer & Company

Cheryl Schwartz

Senior Vice President, U.S. Rare Disease Business Unit Lead and U.S. Commercial Operations

Takeda

Tracey Sikora

Vice President of Research and Clinical Programs

NORD

Stephen Squinto, PhD

Chief Investment Officer

J.P. Morgan Life Science Private Capital

Theresa Strong

Director of Research Programs

Foundation for Prader-Willi Research

David Tischler

Chief Executive Officer

Stealth Mode

Andres Trevino

Global Head of Patient Advocacy

Chiesi

Fumihiko Urano, MD, PhD

Samuel E. Schechter Professor in Medicine

Washington University School of Medicine 

Jian Wang, PhD

Vice President, Digital Innovation

IQVIA R&D