VANISHING WHITE MATTER
Clinical trials for VANISHING WHITE MATTER explained in plain language.
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Single-Patient trial offers hope for untreatable fatal 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 approved 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 …
Matched conditions: VANISHING WHITE MATTER
Phase: EARLY_PHASE1 • Sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre • Aim: Disease control
Last updated Mar 30, 2026 14:32 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: VANISHING WHITE MATTER
Sponsor: AbbVie • Aim: Disease control
Last updated Mar 27, 2026 12:41 UTC