First gene therapy trial for rare brain disorder launches
Disease control
Not yet recruiting
This early-stage trial tests a single dose of FRF-001, a gene therapy injected into the brain fluid, in 12 children and adults with FOXG1 syndrome. The goal is to see if it is safe and can help improve motor skills like sitting or walking. Participants must have a confirmed FOXG1…
Phase: PHASE1, PHASE2 • Sponsor: FOXG1 Research Foundation • Aim: Disease control
Last updated May 17, 2026 16:07 UTC