People with severe hemophilia B are likely to develop hemophilic arthropathy — a painful and degenerative joint disease caused by recurrent bleedings — despite the lower bleeding frequency relative to hemophilia A, a natural history study shows. The data also highlights that prophylactic (preventive) treatment guidelines are not being…
Eloctate can induce immune tolerance in people with hemophilia A more quickly than other regimens, a study suggests. The study, “Real‐world data of immune tolerance induction using recombinant factor VIII Fc fusion protein in patients with severe haemophilia A with inhibitors at high risk for…
A mechanism largely left unexplored was shown to help regulate factor VIII (FVIII) — the clotting protein that is missing or defective in people with hemophilia A — in a study in mice. While early and preclinical work, it is reported to be the first study of this mechanism…
Roctavian, an investigational gene therapy, significantly reduced bleeding rates and the need for other treatments in men with severe hemophilia A over at least one year, top-line data from a Phase 3 clinical trial show. The therapy’s durability in sustaining factor VIII production at levels necessary to…
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…
Parents of children with hemophilia in the U.K. are generally aware of gene therapy, but many lack both the knowledge necessary for an informed decision and confidence regarding this form of treatment, according to a questionnaire and interview-based study. Most of those interviewed were unaware that gene therapies are…
When given as a preventive treatment for nearly six years, Hemlibra (emicizumab) safely and effectively lowered the number of bleeding episodes in patients with severe hemophilia A, according to data from a Phase 1 clinical trial and its long-term extension study. Importantly, reductions in the number of bleeds…
Note: This story was updated Jan. 8, 2021, to clarify that the second set of experiments in mice and non-human primates used a research construct, not the gene therapy. Generation Bio has announced new data showing that its investigational non-viral gene therapy platform can increase levels of the clotting…
Hemophilia News Today brought you daily coverage in 2020 of important discoveries, treatment advancements, clinical trial findings, and other important events related to hemophilia. As we look forward to bringing you more news this year, we present here the 10 most-read stories of 2020, along with a summary of…
The U.S. Food and Drug Administration (FDA) has placed a clinical hold on uniQure’s gene therapy program for hemophilia B, which includes AMT-061 (etranacogene dezaparvovec), due to a possibly related serious adverse event in one patient. The event concerns a preliminary diagnosis of hepatocellular carcinoma (HCC), a form…
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