SANDHOFF DISEASE

Clinical trials for SANDHOFF DISEASE explained in plain language.

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

Gene therapy Follow-Up: 5-Year watch on rare brain diseases

Disease control Ongoing

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…

Matched conditions: SANDHOFF DISEASE

Sponsor: Terence Flotte • Aim: Disease control

Last updated May 15, 2026 11:54 UTC

SANDHOFF DISEASE

Get alerted when new SANDHOFF DISEASE trials appear

Gene therapy Follow-Up: 5-Year watch on rare brain diseases