CREE LEUKOENCEPHALOPATHY
Clinical trials for CREE LEUKOENCEPHALOPATHY explained in plain language.
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Last hope: experimental drug tested on single child with rare brain disease
Disease control OngoingThis study gives one child with Cree Leukoencephalopathy (CLE), a severe and fatal brain disease, access to an experimental drug called fosigotifator. The drug aims to slow or stop the breakdown of the brain's white matter. Researchers will monitor if the benefits outweigh the ri…
Matched conditions: CREE LEUKOENCEPHALOPATHY
Phase: NA • Sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre • Aim: Disease control
Last updated May 26, 2026 07:08 UTC
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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: CREE LEUKOENCEPHALOPATHY
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: CREE LEUKOENCEPHALOPATHY
Sponsor: AbbVie • Aim: Disease control
Last updated May 18, 2026 12:03 UTC