Experimental drug offered as last hope for single patient with devastating brain disease
NCT ID NCT07272525
Summary
This is a compassionate use study for a single patient with Cree Leukoencephalopathy, a rare and fatal brain disease with no current 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 disease. The main goal is to see if the patient survives or avoids needing a ventilator for two years after starting treatment.
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Contacts and locations
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Locations
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McGill University Health Centre
Montreal, Quebec, H4A3J1, Canada
Conditions
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