Gene therapy breakthrough offers hope for boys with rare brain disease
Disease control
Ongoing
This study tests a new gene therapy for young boys with Hunter syndrome, a genetic disorder that damages the brain and body. The therapy uses the child's own blood stem cells, modified to produce the missing enzyme, aiming to stop disease progression. Five boys aged 3 to 22 month…
Phase: PHASE1, PHASE2 • Sponsor: University of Manchester • Aim: Disease control
Last updated May 17, 2026 04:14 UTC