scarlet_sage t1_ixvnm9y wrote
Reply to comment by Sariel007 in A novel medication for hemophilia B has just been approved by the Food and Drug Administration. The treatment is a form of gene therapy, intended to replace a dysfunctional gene that leaves people unable to control their bleeding. by Sariel007
It did not appear to be a cure, though it doesn't explain why.
>It also appeared to lower the rate of bleeding problems by 54% in one trial, and it reduced the need for regular infusions of clotting factors....
>it should reduce the frequency of expensive transfusions; they also say that the drug’s effectiveness is expected to last for several years at least.
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