GLUTARIC ACIDURIA TYPE I

Clinical trials for GLUTARIC ACIDURIA TYPE I explained in plain language.

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New gene therapy hopes to tame rare metabolic disease in kids

Disease control Recruiting now

This early-stage study tests a single-dose gene therapy called VGM-R02b in up to 12 children aged 6 or younger with glutaric acidemia type I, a rare genetic disorder that can cause brain damage. The treatment aims to replace the faulty gene and improve symptoms like movement prob…

Matched conditions: GLUTARIC ACIDURIA TYPE I

Phase: PHASE1 • Sponsor: Shanghai Vitalgen BioPharma Co., Ltd. • Aim: Disease control

Last updated May 17, 2026 08:53 UTC

GLUTARIC ACIDURIA TYPE I

Get alerted when new GLUTARIC ACIDURIA TYPE I trials appear

New gene therapy hopes to tame rare metabolic disease in kids