TAY-SACHS DISEASE

Clinical trials for TAY-SACHS DISEASE explained in plain language.

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Trials to join now! 4 Not yet finished but already full! 1 Completed 3 Terminated 2

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…

Matched conditions: TAY-SACHS DISEASE

Sponsor: Terence Flotte • Aim: Disease control

Last updated Jun 27, 2026 07:54 UTC

TAY-SACHS DISEASE

Get alerted when new TAY-SACHS DISEASE trials appear

Gene therapy for rare brain diseases: 5-Year safety check begins