AADC DEFICIENCY

Clinical trials for AADC DEFICIENCY explained in plain language.

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Brain surgery delivers gene therapy to children with rare neurological disorder

Disease control Ongoing

This study is testing the safety of a new surgical tool and a one-time gene therapy for children with Aromatic L-amino Acid Decarboxylase (AADC) deficiency, a rare genetic disorder that severely affects brain development and movement. The therapy, called eladocagene exuparvovec, …

Matched conditions: AADC DEFICIENCY

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

Last updated Apr 02, 2026 07:26 UTC

AADC DEFICIENCY

Get alerted when new AADC DEFICIENCY trials appear

Brain surgery delivers gene therapy to children with rare neurological disorder