Recently, Novo Nordisk announced that Rebinyn (Coagulation Factor IX (Recombinant), pegylated), was 1 of 5 drugs that will be available for patients in Canada with hemophilia B. Making those drugs available in Canada was dependent on patients’ input into the value those treatments brings to the hemophilia B community.
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The results of a phase 1/2, clinical study looking at the safety and efficacy of gene therapy to treat 9 adults with severe hemophilia A was recently published in the New England Journal of Medicine (NEJM). And the results are very promising.
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This week in the New England Journal of Medicine, Lindsey George, MD and colleagues report on their Phase 1/2 study assessing the safety and efficacy of gene therapy to treat patients with hemophilia B. And the results are very impressive.
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