AADC DEFICIENCY

Clinical trials for AADC DEFICIENCY explained in plain language.

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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