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Gene therapy trial targets Huntington's disease in 5 patients

NCT ID NCT05541627

First seen Nov 01, 2025 · Last updated May 20, 2026 · Updated 31 times

Summary

This early-stage trial tests a gene therapy called AB-1001 in 5 adults with early Huntington's disease. The therapy is injected directly into the brain to try to slow the disease. The main goal is to check safety and side effects, while also looking at brain changes on MRI scans.

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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

Locations

  • Institut du Cerveau (ICM), Hôpital La Pitié Salpêtrière APHP

    Paris, Île-de-France Region, 75013, France

Conditions

Explore the condition pages connected to this study.