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 rare childhood movement disorder

Disease control Ongoing

This trial tests a gene therapy called VGN-R09b for children aged 1.5 to 8 years with severe AADC deficiency, a rare genetic disorder that severely limits movement. The treatment is injected directly into a brain region called the putamen. The study aims to see if it is safe and …

Matched conditions: AROMATIC L-AMINO ACID DECARBOXYLASE DEFICIENCY

Phase: PHASE2, PHASE3 • Sponsor: Shanghai Vitalgen BioPharma Co., Ltd. • Aim: Disease control

Last updated May 04, 2026 16:23 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 rare childhood movement disorder