Most people with hemophilia A or B who switched from an approved prophylactic (preventive) therapy regimen to once-monthly treatment with fitusiran in the ATLAS-PPX clinical trial had no bleeds over seven months on the investigational therapy, new data show. Fitusiran also reduced bleeding rates regardless of the presence…
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Last week, my wife, Cazandra, and I attended the National Bleeding Disorders Foundation’s (NBDF) annual symposium. As parents of two adult sons who have severe hemophilia A, or factor VIII deficiency, we’re familiar with the lay of the land. The conference provided a space for us to discover…
Hemophilia is a bleeding disorder caused by a mutation in genes that provide instructions for blood clotting factors — the proteins that help in blood clotting. Mutations in the genes for clotting factors VIII, IX, and XI result in their deficiency, causing hemophilia A, hemophilia B, and hemophilia C, respectively.
Caeleb, the child doctors said I’d never have, has changed my life. He is 16 and thinking about his future. That may sound like a normal thing for a 16-year-old, but I was unsure about his future with his hemophilia A. His life with this disease has been…
Infection by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the coronavirus causing COVID-19, can induce the onset of acquired hemophilia A (AHA), a case report has found. As such, blood coagulation should be monitored closely in patients hospitalized for COVID-19, scientists said. The study, “…
An experimental gene therapy using modified blood stem cells helped five adults with severe hemophilia A attain therapeutic levels of clotting factor VIII (FVIII) to prevent and control bleeding, according to a small first-in-human study. Over the course of the Phase 1 clinical study (NCT05265767), none of the…
Using prednisone in combination therapies increases an infection risk but also better raises the likelihood of patients with acquired hemophilia A (AHA) achieving complete remission than does prednisone given alone, a Dutch study found. The retrospective study, which evaluated patients diagnosed over a 27-year period, also found that…
Treatment with Hemlibra (emicizumab) improved joint health in people with hemophilia A participating in the Phase 3 HAVEN 3 trial, with the most pronounced improvements seen in those younger than 40 or in patients who had joints with frequent bleeds. Findings were detailed in the study “…
BAY 94-9027, developed by Bayer, is an investigational treatment for severe hemophilia A. It is a PEGylated molecule of recombinant factor VIII in which the B-domain, a region within the factor, is deleted. The therapy is produced using a baby hamster kidney cell line (BHK-21), and has…
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What I Want for My Future Child
Time flies so surprisingly fast. It just dawned on me that I’ll be a father in three months; my wife, Cza, is already 26 weeks into her pregnancy. There’s a lot of pressure, but I hope that my kid can be proud to have me as a father. It’s of…