Gene therapy hope for rare childhood disease AGU
Disease control
Not yet recruiting
This early-stage trial tests a single dose of a gene therapy called DANAGALEX in 9 adults and children with aspartylglucosaminuria (AGU), a rare genetic disorder. The goal is to see if the treatment is safe and can reduce harmful substances in the body. Researchers will monitor s…
Phase: PHASE1, PHASE2 • Sponsor: Rare Trait Hope • Aim: Disease control
Last updated Jun 27, 2026 12:07 UTC