Gene therapy breakthrough? new trial targets rare brain disorder FOXG1
Disease control
ENROLLING_BY_INVITATION
This early-stage trial tests a new gene therapy called FRF-001 for FOXG1 syndrome, a rare genetic disorder that affects brain development. The therapy is given as a single injection into the brain's fluid spaces. The study will include 12 children and adults and will check for sa…
Phase: PHASE1, PHASE2 • Sponsor: FOXG1 Research Foundation • Aim: Disease control
Last updated Jul 26, 2026 00:00 UTC