CREE LEUKOENCEPHALOPATHY
Clinical trials for CREE LEUKOENCEPHALOPATHY explained in plain language.
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Experimental drug offered as last hope for single patient with devastating brain disease
Disease control OngoingThis is a compassionate use study for a single patient with Cree Leukoencephalopathy, a rare and fatal brain disease with no current treatments. The study will provide access to an experimental drug called Fosigotifator (FGT) to see if it can slow or stop the progression of the d…
Matched conditions: CREE LEUKOENCEPHALOPATHY
Phase: EARLY_PHASE1 • Sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre • Aim: Disease control
Last updated Apr 03, 2026 23:50 UTC
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Experimental drug offered for devastating rare brain diseases
Disease control NO_LONGER_AVAILABLEThis program provided early access to an investigational drug called fosigotifator for people with vanishing white matter disease and Cree leukoencephalopathy—two rare, serious brain disorders. It was designed to make the treatment available to eligible patients before it receive…
Matched conditions: CREE LEUKOENCEPHALOPATHY
Sponsor: AbbVie • Aim: Disease control
Last updated Mar 27, 2026 12:41 UTC
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Hope for a single child: groundbreaking drug trial for fatal brain disease
Disease control OngoingThis study provides a single patient with a rare, fatal brain disease access to an experimental drug called fosigotifator. The goal is to see if the drug can slow or stop the breakdown of brain tissue, potentially improving symptoms and quality of life. The patient has no other t…
Matched conditions: CREE LEUKOENCEPHALOPATHY
Phase: NA • Sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre • Aim: Disease control
Last updated Mar 27, 2026 12:41 UTC