Brain-Injected gene therapy offers hope for devastating genetic disorder
Disease control
Not yet recruiting
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 thera…
Phase: PHASE1, PHASE2 • Sponsor: FOXG1 Research Foundation • Aim: Disease control
Last updated Mar 30, 2026 14:31 UTC