Experimental drug offers hope for children with devastating brain disorder
NCT ID NCT07300397
First seen Jan 11, 2026 · Last updated May 08, 2026 · Updated 16 times
Summary
This study gives one child with Cree leukoencephalopathy (CLE) access to an experimental drug called fosigotifator. CLE is a rare, inherited brain disease that destroys white matter and leads to early death. The drug targets the genetic cause of the disease and may slow or stop brain damage, reduce symptoms, and improve quality of life.
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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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