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 […]
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Following a positive opinion from the European Medicines Agency (EMA), the transfer of marketing authorization of Strimvelis from Orchard Therapeutics has been approved by the European Commission. Fondazione Telethon (Italy) will now be responsible for providing the gene therapy to eligible patients in the European Union. Fondazione Telethon, one of the main Italian biomedical charities, […]
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