Gene therapy Follow-Up: 5-Year watch on rare brain diseases
NCT ID NCT06614569
First seen Oct 31, 2025 · Last updated May 14, 2026 · Updated 26 times
Summary
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 to understand how safe and effective the therapy is over time.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Locations
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UMass Chan Medical School
Worcester, Massachusetts, 01655, United States
Conditions
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