Gene therapy hope for rare AGU disease enters human trials
Disease control
Not yet recruiting
This early-stage trial tests a single-dose gene therapy called DANAGALEX in 9 adults and children with AGU, a rare genetic disorder. The goal is to see if it is safe and can reduce harmful substances in the body. Participants must have a confirmed AGU diagnosis and be enrolled in…
Phase: PHASE1, PHASE2 • Sponsor: Rare Trait Hope • Aim: Disease control
Last updated May 12, 2026 13:41 UTC