AADC DEFICIENCY

Clinical trials for AADC DEFICIENCY explained in plain language.

Never miss a new study

Get alerted when new AADC DEFICIENCY trials appear

Sign up with your email to follow new studies for AADC DEFICIENCY, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.

Our safety recommendation!

By submitting, you agree to our Terms of use

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

Brain gene therapy delivery system tested in kids with rare disorder

Disease control Ongoing

This study tests a special needle (SmartFlow cannula) designed to safely deliver a gene therapy drug (eladocagene exuparvovec) into the brains of 13 children with AADC deficiency, a rare genetic disorder that affects movement and development. The main goals are to check if the pr…

Matched conditions: AADC DEFICIENCY

Phase: PHASE2 • Sponsor: PTC Therapeutics • Aim: Disease control

Last updated Jun 27, 2026 12:23 UTC

AADC DEFICIENCY

Get alerted when new AADC DEFICIENCY trials appear

Brain gene therapy delivery system tested in kids with rare disorder