You are not alone. Coping with hemophilia as a patient, caretaker, or medical provider can seem overwhelming at times. When I am overwhelmed, have a question, or just want to talk to someone who I know will “get it,” I reach out to my “blood sisters” and…

At standard U.S. doses, Hemlibra (emicizumab) provides comparable or even superior clinical benefits than prophylactic factor VIII (FVIII) replacement therapy among adults with severe hemophilia A and no inhibitors, according to a report from the Institute for Clinical and Economic Review (ICER). Hemlibra is also cost-saving, due to…

CSL Behring’s rVIII-SingleChain, a novel recombinant Factor VIII (rFVIII) treatment candidate, recently demonstrated effectiveness in surgery and in the control of bleeding in patients with severe hemophilia A. The Phase 1 to Phase 3 clinical trial results, recently published in the journal Blood, also demonstrated low annualized bleeding rates in patients…

I’ve lost count of how many times I’ve seen phrases like “victims of hemophilia” or “afflicted with hemophilia” in local news articles. Sometimes the phrase is “suffering from hemophilia.” To many readers, these words might sound sympathetic — even caring. But to those of us…

Knowledge about the clinical benefits of treatments and their importance in the outcome of patients, as well as more stable therapies, will likely improve treatment adherence among hemophilia patients in the U.S., according to new research. The study, “Treatment adherence in hemophilia,” was conducted by Dr. Courtney Thornburg, medical…

A single infusion of the experimental gene therapy GS001 safely increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A  — for almost three years, effectively reducing bleeds and the need for replacement therapies at doses much lower than those used in currently approved…

Being unemployed or under-educated can have a negative effect on hemophilia patients’ quality of life, including their pain levels and ability to function, according to the results of a questionnaire that were presented at the ASH Annual Meeting and Exposition. In the patients surveyed, increased pain, functional impairment and reduced…

Four years after treatment with the gene therapy Hemgenix (etranacogene dezaparvovec), most people with hemophilia B aren’t taking preventive therapies and annual bleeding rates remain substantially reduced, new data from the Phase 3 HOPE-B trial shows. CSL Behring, which markets Hemgenix, presented the results at the 18th…

Data from ongoing Phase 3 clinical trials show that Genentech’s Hemlibra (emicizumab-kxwh) prophylaxis — or preventive treatment — greatly outperforms current standard-of-care therapies, leading to significant reductions in the number of bleeds in hemophilia A patients regardless of inhibitor status. Promising results from these pivotal studies may lead…