Gene therapy for rare brain diseases: 5-Year safety check begins
Disease control
Ongoing
This study follows 7 people who previously received a gene therapy called AXO-AAV-GM2 for Tay-Sachs or Sandhoff disease — rare, fatal genetic disorders that destroy nerve cells. Researchers will track them for up to 5 years to see if the treatment remains safe and whether it help…
Sponsor: Terence Flotte • Aim: Disease control
Last updated Jun 27, 2026 07:54 UTC