VANISHING WHITE MATTER
Clinical trials for VANISHING WHITE MATTER explained in plain language.
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Desperate patients get early access to experimental brain drug
Disease control NO_LONGER_AVAILABLEThis program offers early access to fosigotifator, an oral drug, for people with Vanishing White Matter or Cree Leukoencephalopathy—two rare, serious brain diseases. A doctor must decide if the potential benefit outweighs the risks for each patient. The drug is not yet approved, …
Matched conditions: VANISHING WHITE MATTER
Sponsor: AbbVie • Aim: Disease control
Last updated Jun 27, 2026 08:11 UTC
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Last hope: experimental drug tested in single child with rare brain disease
Disease control OngoingThis study gives one patient with Cree Leukoencephalopathy, a rare and fatal brain disease, access to an experimental drug called fosigotifator. The drug aims to slow or stop the brain damage that causes severe disability and early death. Researchers will track whether the patien…
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 Jun 27, 2026 08:07 UTC