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Fondazione Telethon announces submission of MAA to EMA for Gene Therapy for the treatment of Wiskott-Aldrich Syndrome patients

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.


Sekretariat der DG-GT e.V.
Institut für Experimentelle Hämatologie
Hildegard Büning
Carl-Neuberg-Str. 1
30625 Hannover

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© 2021 Die Deutsche Gesellschaft für Gentherapie e.V.

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