Genterapi mot blödarsjuka visade effekt ännu efter fyra år

Den omtalade genterapin Hemgenix mot hemofili B visar god effekt fortfarande fyra år efter att behandlingen utförts.

By Anders Göransson

February 12, 2025 11:56 AM

Hemgenix (etranakogen-dezaparvovek) fick ett villkorat godkännande från den europeiska läkemedelsmyndigheten EMA 2022, och har nu utvärderats i en uppföljningsstudie. I den kunde 94 procent av patienterna fyra år efter engångsbehandlingen klara sig utan de kontinuerliga intravenösa infusioner av faktor IX-profylax som är standardbehandling vid sjukdomen.

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