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Last hope: experimental drug tested in single patient with rare fatal brain disorder

NCT ID NCT07272525

First seen Jan 04, 2026 · Last updated May 19, 2026 · Updated 22 times

Summary

This study provides an experimental drug called fosigotifator to one patient with Cree Leukoencephalopathy, a rare and fatal brain disease that currently has no treatments. The drug aims to slow or stop brain damage and improve quality of life. The patient must be in early stages of the disease.

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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

  • McGill University Health Centre

    Montreal, Quebec, H4A3J1, Canada

Conditions

Explore the condition pages connected to this study.