VANISHING WHITE MATTER
Clinical trials for VANISHING WHITE MATTER explained in plain language.
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Last hope: experimental drug given to single child with fatal brain disorder
Disease control OngoingThis study provides an experimental drug called fosigotifator to one patient with Cree Leukoencephalopathy, a rare and deadly brain disease that currently has no treatments. The goal is to see if the drug can slow or stop the brain damage and improve quality of life. The patient …
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 11, 2026 20:37 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 Apr 30, 2026 15:49 UTC