, the first gene therapy to treat adults with haemophilia B, a genetic bleeding disorder resulting from missing or insufficient levels of blood-clotting Factor IX.
In a study Hemgenix, distributed by CSL Behring, cut the number of bleeding events expected over a year by 54%. It also removed the need for 94% of patients to receive infusions of Factor IX, saving them considerable time and money. "Today's approval provides a new treatment option for patients with haemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of haemophilia."reported, making it the world's most expensive medicine by some distance.
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