Fondazione Telethon

This study tests a gene therapy for people with Wiskott-Aldrich syndrome, a rare genetic disease that weakens the immune system and causes bleeding. The treatment uses the patient's own blood stem cells, which are modified in a lab to fix the faulty gene and then returned to the …

This study follows 50 people with ADA-SCID, a severe immune disorder, who received Strimvelis gene therapy. Researchers will monitor participants for years to check for side effects, immune function, and overall health. The goal is to understand the long-term safety and how well …