Last hope: experimental drug given to single child with fatal brain disorder
NCT ID NCT07272525
First seen Jan 04, 2026 · Last updated May 10, 2026 · Updated 21 times
Summary
This study provides an experimental drug called fosigotifator to one patient with Cree Leukoencephalopathy, a rare and deadly brain disease that currently has no treatments. The goal is to see if the drug can slow or stop the brain damage and improve quality of life. The patient must have a confirmed diagnosis and be in the early stages of 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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Locations
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McGill University Health Centre
Montreal, Quebec, H4A3J1, Canada
Conditions
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