New hope for rare brain disease: expanded access to experimental drug zilganersen
NCT ID NCT07487389
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This program offers early access to zilganersen for people with Alexander disease, a rare genetic brain disorder. It is for US residents aged 2 and older who have not responded to or cannot tolerate approved treatments. The goal is to provide the drug while it is still being studied.
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Conditions
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