Gene Therapy for Hemophilia B Shows Promise - Managed Healthcare Executive

A small study finds that an adeno-associated virus gene therapy eliminated both spontaneous bleeding and the need for factor IX prophylaxis in patients with hemophilia B.

A gene therapy being tested in a phase 1/2 study for hemophilia B achieved normal levels of factor IX at low doses, but required immunosuppression, according to findings of a study recently published… [+3568 chars]

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