Fondazione Telethon
Clinical trials sponsored by Fondazione Telethon, explained in plain language.
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Gene therapy aims to free Beta-Thalassemia patients from lifelong transfusions
Disease control Recruiting nowThis study tests a single-dose gene therapy for people with transfusion-dependent beta-thalassemia, a blood disorder that requires regular red blood cell transfusions. The therapy uses a patient's own stem cells, modified with a virus to produce healthy hemoglobin, and then infus…
Phase 2 • Sponsor: Fondazione Telethon • Aim: Disease control
Last updated Jul 29, 2026 00:00 UTC
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Gene therapy offers new hope for kids with brittle bone disease
Disease control Recruiting nowThis 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 1/2 • Sponsor: Fondazione Telethon • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC