Hope for a single child: groundbreaking drug trial for fatal brain disease
NCT ID NCT07300397
Summary
This study provides a single patient with a rare, fatal brain disease access to an experimental drug called fosigotifator. The goal is to see if the drug can slow or stop the breakdown of brain tissue, potentially improving symptoms and quality of life. The patient has no other treatment options for this condition, which is caused by a specific genetic change.
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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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