Brain gene therapy offers hope for kids with rare movement disorder
Disease control
Recruiting now
This early-stage study tests a gene therapy for children with AADC deficiency, a rare genetic disorder that causes severe movement problems. The therapy delivers a working gene directly to the brain to help produce important brain chemicals. The study aims to see if the treatment…
Phase: PHASE1 • Sponsor: Krzysztof Bankiewicz • Aim: Disease control
Last updated May 04, 2026 16:32 UTC