First gene therapy for rare brain disorder begins testing in kids
Disease control
Recruiting now
This study tests a new gene therapy for children aged 2-12 with CTNNB1 neurodevelopmental syndrome, a rare genetic disorder causing severe developmental delays. Twelve participants will receive a single dose of the therapy directly into the brain fluid, along with lifelong immune…
Phase: PHASE1, PHASE2 • Sponsor: CTNNB1 Foundation • Aim: Disease control
Last updated May 26, 2026 12:02 UTC