AADC DEFICIENCY

Clinical trials for AADC DEFICIENCY explained in plain language.

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Trials to join now! 1 Not yet finished but already full! 1

Brain gene therapy offers hope for kids with rare movement disorder

Disease control Recruiting now

This study tests a one-time gene therapy for children with AADC deficiency, a rare genetic disorder that causes severe movement problems and developmental delays. The therapy delivers a working gene directly to the brain using a harmless virus. The goal is to improve motor functi…

Matched conditions: AADC DEFICIENCY

Phase: PHASE1 • Sponsor: Krzysztof Bankiewicz • Aim: Disease control

Last updated May 15, 2026 11:55 UTC

AADC DEFICIENCY

Get alerted when new AADC DEFICIENCY trials appear

Brain gene therapy offers hope for kids with rare movement disorder