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

New gene therapy trial offers hope for kids with rare brain disorder

Disease control Ongoing

This study tests a gene therapy called eladocagene exuparvovec in 13 children with AADC deficiency, a rare genetic disorder that affects brain function. The therapy is given directly into the brain using a special needle. The main goals are to check safety and see if it raises le…

Matched conditions: AADC DEFICIENCY

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

Last updated May 11, 2026 20:38 UTC

AADC DEFICIENCY

Get alerted when new AADC DEFICIENCY trials appear

New gene therapy trial offers hope for kids with rare brain disorder