2025 RARE Drug Development Symposium
Expert Office Hours
Get one-to-one time with experts on topics such as data collection, research strategy, therapy development, and more.
Expert Office Hours
Wednesday, September 3 from 4:45pm – 5:30pm
Thursday, September 4 from 12:00pm – 1:00pm
*not all experts are available for all office hours
Get answers to your questions by scheduling a one-to-one
15-minute session with one of our experts on topics such as
data collection, research strategy, therapy development,
biorepositories, and clinical trial readiness.
Attendees are permitted to register for only one Expert Office Hour per day
to ensure equitable access for all participants.
Additional appointments may be booked closer to the start of the symposium if available.
Raymond Belanger Deloge, MS, CGC
Genetic Counselor
Rosamund Stone Zander Translational Neuroscience Center
Boston Children’s Hospital
Elizabeth Buttermore, Ph.D.
Director, Translational In Vitro Models
Boston Children’s Hospital
Ebony Dashiell-Aje, Ph.D.
Executive Director and Head, Patient-Centered Outcomes Science
BioMarin Pharmaceutical
Wendy Erler
Senior Vice President
Sarepta Therapeutics
Susan Faja, Ph.D.
Associate Professor, Pediatrics
Boston Children’s Hospital
Cassi Friday, Ph.D.
Director of Research
Cure HHT
Sarah Gladstone, M.D.
Chief Scientific Officer, Chief Medical Officer
The Snow Foundation for Wolfram Syndrome
Rob Goldstein-Mahon. MSPA
Director, Patient Advocacy
Ionis
Amy Grover
Executive Director of Patient Advocacy
Catalyst Pharmaceuticals
Joe Katakowski, Ph.D.
Director, Rare Diseases
RTW Foundation
Walt Kowtoniuk Ph.D.
Venture Partner
Third Rock Venture
Craig Lipset
Co-Founder and Co-Chair of the Decentralized Trials & Research Alliance
Nicole Litt
Relationship Coordinator, Rare Disease
RTW Foundation
Steven Roberds, Ph.D.
Chief Scientific Officer
TSC Alliance
Leora Schiff, MS, MBA
Principal
Altius Strategy Consulting
Karmen Trzupek, MS, CGC
Senior Director of Scientific Programs
Global Genes
Krista Vasi, M.P.A
Executive Director
Usher Syndrome Coalition
Paul Wang, M.D.
Senior Scientific Director
SFARI (Simons Foundation)
