Experimental gene therapy offers hope for kids with rare brain disorder
Disease control
Recruiting now
This early-stage study tests a gene therapy for children with AADC deficiency, a rare genetic disorder that affects brain chemicals needed for movement and mood. The therapy delivers a working gene directly into the brain using a virus. The goal is to see if it is safe and can im…
Phase: PHASE1 • Sponsor: Krzysztof Bankiewicz • Aim: Disease control
Last updated Jun 02, 2026 11:57 UTC