Fondazione Telethon is proud to announce that it has submitted the Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the gene therapy - etuvetidigene autotemcel - for the treatment of Wiskott-Aldrich Syndrome (WAS) patients
If approved, the gene therapy will provide a valid treatment option, with excellent results in terms of both safety and efficacy in WAS patients for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.