Brain gene therapy trial offers hope for kids with rare movement disorder
Disease control
Recruiting now
This early-stage trial tests a gene therapy called AAV2-hAADC in 42 children with AADC deficiency, a rare genetic disorder that causes severe movement problems and developmental delays. The therapy delivers a working gene directly into brain areas that control movement, aiming to…
Phase: PHASE1 • Sponsor: Krzysztof Bankiewicz • Aim: Disease control
Last updated Jul 15, 2026 00:00 UTC