Brain gene infusion offers hope for kids with rare movement disorder
Disease control
Recruiting now
This study tests a gene therapy for children with AADC deficiency, a rare genetic disorder that causes severe movement problems. The treatment delivers a working gene directly to the brain using a virus. The goal is to see if it is safe and can improve symptoms like muscle contro…
Phase: PHASE1 • Sponsor: Krzysztof Bankiewicz • Aim: Disease control
Last updated May 25, 2026 08:57 UTC