Gene therapy offers new hope for kids with brittle bone disease
Disease control
Recruiting now
This study tests a new gene therapy for children with a severe inherited bone disease called osteopetrosis. The therapy uses the child's own blood stem cells, modified to fix the genetic defect, and then returned to the body. The goal is to improve blood cell production and survi…
Phase: PHASE1, PHASE2 • Sponsor: Fondazione Telethon • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC