One-Time brain surgery offers hope for cure in devastating childhood disease
⭐️ CURE ⭐️
Recruiting now
This study is testing a one-time gene therapy for children with AADC deficiency, a rare genetic disorder that severely impairs brain development and movement. Doctors use precise MRI-guided surgery to deliver a corrective gene directly into a specific part of the brain. The main …
Phase: PHASE1 • Sponsor: Krzysztof Bankiewicz • Aim: ⭐️ CURE ⭐️
Last updated Mar 31, 2026 12:12 UTC