Aromatic L-amino acid decarboxylase deficiency

Clinical trials for Aromatic L-amino acid decarboxylase deficiency explained in plain language.

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Gene therapy injection shows promise for kids with rare movement disorder

Disease control Ongoing

This trial tests a gene therapy called VGN-R09b for children with severe AADC deficiency, a rare genetic disorder that affects movement and brain function. The therapy is injected directly into the brain. The study aims to see if it is safe and helps children achieve motor milest…

Matched conditions: AROMATIC L-AMINO ACID DECARBOXYLASE DEFICIENCY

Phase 2/3 • Sponsor: Shanghai Vitalgen BioPharma Co., Ltd. • Aim: Disease control

Last updated Jun 27, 2026 12:23 UTC

Aromatic L-amino acid decarboxylase deficiency

Get alerted when new Aromatic L-amino acid decarboxylase deficiency trials appear

Gene therapy injection shows promise for kids with rare movement disorder