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