Brain-Injected gene therapy offers hope for devastating genetic disorder

NCT ID NCT07293546

Summary

This is the first human study testing FRF-001, a gene therapy delivered directly to the brain, for people with FOXG1 syndrome. The trial will enroll 12 children and adults to check if the treatment is safe and if it helps improve movement skills. Researchers will inject the therapy into the fluid around the brain and monitor participants closely for side effects and potential benefits.

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Contacts and locations

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Conditions

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