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 early-stage study tests a gene therapy for children with AADC deficiency, a rare genetic disorder that causes severe movement problems. The therapy delivers a working gene directly to the brain to help produce important brain chemicals. The study aims to see if the treatment…

Matched conditions: AADC DEFICIENCY

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

Last updated May 04, 2026 16:32 UTC

AADC DEFICIENCY

Get alerted when new AADC DEFICIENCY trials appear

Brain gene therapy offers hope for kids with rare movement disorder