FDA approves Hemgenix, the first gene therapy to treat adults with hemophilia B

FDA approves Hemgenix, the first gene therapy to treat adults with hemophilia B

The U.S. Food and Drug Administration (FDA) has approved Hemgenix (etranacogene dezaparvovec) gene therapy for the treatment of adults with hemophilia B (congenital factor 9 deficiency) who are currently using prophylactic factor 9 therapy; have a current or historical life-threatening haemorrhage; or have severe and repeated spontaneous bleeding episodes. Despite advances in the treatment of …

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