Gene therapy injection targets Huntington's disease in small trial
NCT ID NCT05541627
First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 30 times
Summary
This early-stage trial tests a gene therapy called AB-1001, injected directly into the brain, in 5 adults with early Huntington's disease. The main goal is to check safety and side effects, while also looking at brain changes on MRI. It is not a cure, but aims to slow or control the disease.
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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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Institut du Cerveau (ICM), Hôpital La Pitié Salpêtrière APHP
Paris, Île-de-France Region, 75013, France
Conditions
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