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US regulators approved CSL Behring’s haemophilia B gene therapy, a one-off infusion that frees patients from regular treatments but costs $3.5m a dose, making it the most expensive medicine in the world.
New drugs such as emicizumab (Hemlibra) are transforming the lives of patients with hemophilia A, and more are on their way. "It's an amazing time to be a hemophilia provider," says Alice D. Ma, MD.
New approaches to treating hemophilia may soon replace the decades-old approach of using factor concentrates to control bleeding in patients with hemophilia A and hemophilia B.