Experimental gene therapy targets rare fatal brain diseases in kids
NCT ID NCT04669535
First seen Nov 01, 2025 · Last updated May 12, 2026 · Updated 29 times
Summary
This study tested a gene therapy called AXO-AAV-GM2 in children with Tay-Sachs or Sandhoff disease, rare and fatal genetic disorders that damage the brain. The therapy aimed to deliver healthy genes to brain cells to restore a missing enzyme. The trial enrolled up to 9 children aged 6 months to 12 years, but was terminated early. The main goal was to check safety and find the right dose.
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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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Massachusetts General Hospital, Center for Rare Neurological Diseases
Boston, Massachusetts, 02114, United States
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University of Massachusetts Medical Health Center
Worcester, Massachusetts, 01655, United States
Conditions
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