This study follows 7 people with Tay-Sachs or Sandhoff disease who previously received a gene therapy called AXO-AAV-GM2. Researchers will track their health for up to 5 years to check for long-term side effects and see if the treatment slows the disease's progress. The goal is t…
Sponsor: Terence Flotte • Aim: Disease control
Last updated May 15, 2026 11:54 UTC