Gene therapy watch: 5-Year Follow-Up for rare brain diseases begins
NCT ID NCT06614569
First seen Oct 31, 2025 · Last updated May 19, 2026 · Updated 27 times
Summary
This study checks on 7 people with Tay-Sachs or Sandhoff disease who received a gene therapy called AXO-AAV-GM2. Researchers will watch for side effects and see how the treatment affects their thinking, movement, and daily life over 5 years. The goal is to learn if the therapy is safe and helps control these serious genetic conditions.
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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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Contacts and locations
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Locations
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UMass Chan Medical School
Worcester, Massachusetts, 01655, United States
Conditions
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