FDA approves first gene therapy to treat adults with Hemophilia B

FDAToday, the U.S. Food and Drug Administration approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.


from Breaking World Pharma News https://ift.tt/vlNpRZ4

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