IRDiRC Funders Constituent Committee (FCC) Member Fondazione Telethon is pleased to announce a new discovery from San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), which reveals the key liver cells driving organ growth and advancing pediatric gene therapy. Published in the Journal of Hepatology, the study reveals that a minority of liver cells in newborns […]
Read MoreFondazione Telethon announced that it has submitted the Marketing Authorization Application (MAA) for the gene therapy – etuvetidigene autotemcel – for the treatment of patients with Wiskott-Aldrich Syndrome (WAS), a rare genetic disease of the immune system to the European Medicines Agency (EMA). If approved, etuvetidigene autotemcel will provide a valid treatment option, with excellent results in terms of both safety […]
Read More