VANISHING WHITE MATTER
Clinical trials for VANISHING WHITE MATTER explained in plain language.
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Last hope: experimental drug tested in single patient with rare fatal brain disorder
Disease control OngoingThis study provides an experimental drug called fosigotifator to one patient with Cree Leukoencephalopathy, a rare and fatal brain disease that currently has no treatments. The drug aims to slow or stop brain damage and improve quality of life. The patient must be in early stages…
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 May 19, 2026 11:48 UTC
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Desperate families get early access to experimental brain disease drug
Disease control NO_LONGER_AVAILABLEThis program offers early access to the investigational drug fosigotifator for people with vanishing white matter or Cree leukoencephalopathy, rare and serious brain diseases. It is not a clinical trial but a way for eligible patients to receive the drug before it is officially a…
Matched conditions: VANISHING WHITE MATTER
Sponsor: AbbVie • Aim: Disease control
Last updated May 18, 2026 12:03 UTC