Fondazione Telethon
Clinical trials sponsored by Fondazione Telethon, explained in plain language.
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Gene therapy for 'Bubble Boy' disease under Long-Term watch
Disease control ENROLLING_BY_INVITATIONThis study follows about 50 people with a rare immune disorder called ADA-SCID who have received a gene therapy treatment called Strimvelis. The goal is to track their health for many years to see if the treatment remains safe and effective. Researchers will monitor for side effe…
Sponsor: Fondazione Telethon • Aim: Disease control
Last updated Jun 27, 2026 12:36 UTC
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Gene therapy aims to stop severe infections in rare immune disorder
Disease control OngoingThis trial tests a gene therapy called OTL-103 for people with Wiskott-Aldrich syndrome, a rare genetic disorder that weakens the immune system and causes bleeding. The therapy uses the patient's own blood stem cells, modified in a lab to carry a working copy of the faulty gene, …
Phase: PHASE3 • Sponsor: Fondazione Telethon • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC