Leading the worldwide fight to treat and cure
Tay-Sachs, Canavan, GM1 and Sandhoff diseases

NTSAD Virtual Family Conference | 2021 GM1 Gangliosidosis Research Breakout

Manolo Belotto, PhD, General Manager and President
Gain Therapeutics

Jose Alcantara, Project Scientist
Idorsia Pharmaceuticals natural history study

The video recording containing Idorsia's slides cannot be publicly shared at this time. Please contact This email address is being protected from spambots. You need JavaScript enabled to view it., Director of Family Services, regarding any questions you may have about their natural history study.

Olivier Morand, CEO
Azafaros drug development program

Samantha Parker, Chief Patient Access Officer
Lysogene gene therapy program

David Weinstein, MD, MMSc, Passage Bio and Jeanine Jarnes, PharmD, U of MN
Passage Bio gene therapy clinical trial

Gavin Corcoran, Chief R&D Officer 
NIH / Sio gene therapy clinical trial

Virtual Conference

View the NTSAD Annual Family Conference Research Exhibit Hall

Please contact Becky Benson, Conference and Family Services Coordinator here with any questions or concerns that are conference related.

2021 Conference Sponsors

Laura and Simeon Schindelman
Bonnie and Barry Davis in loving memory of Adam Davis 
The Evan Lee Ungerleider Fund
NTSAD New York Area Fund 
*sponsors as of 4/15/21