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AADC DEFICIENCY

Clinical trials for AADC DEFICIENCY explained in plain language.

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  • 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 …

    Matched conditions: AADC DEFICIENCY

    Phase: PHASE1 • Sponsor: Krzysztof Bankiewicz • Aim: ⭐️ CURE ⭐️

    Last updated Mar 31, 2026 12:12 UTC

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