In keeping with its mission to change how members of the bleeding disorder community think about their condition, Octapharma USA has joined with GutMonkey to provide a challenging week-long, water-based experience aimed at boosting patient confidence in managing disease. With an Octapharma grant, 10 participants in “Leading X…
A liver transplant can be live-saving for hemophilia patients, but may not always fully cure the disease, according to the case report about a hemophilia A patient. The letter to the editor describing the report, “Orthotopic liver transplantation for haemophilia A may not…
The investigational gene therapy SB-525 is tolerated well by patients, and dose-dependent responses are positive, according to preliminary results of a Phase 1/2 clinical trial of hemophilia A. The potential gene therapy is being developed as a partnership between Sangamo Therapeutics and Pfizer. It consists of…
About a third of European and North American hemophilia patients are overweight or obese, which is associated with increased joint dysfunction and chronic pain, according to a recent review study. The review, “Obesity in the global haemophilia population: prevalence, implications and expert opinions for weight management,”…
Idelvion (albutrepenonacog alfa), a hemophilia B treatment approved for up to 14-day dosing, is now available in a large, 3500 IU vial size (3500 IU),…
Hemlibra (emicizumab) was able to prevent excessive bleeding in patients with severe hemophilia A without inhibitors, according to results from a Phase 3 trial. Trial findings were published in the study, “Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors,” in The New England Journal of…
The U.S. Food and Drug Administration has approved Bayer’s Jivi, previously known as BAY94-9027, as a preventive therapy for hemophilia A in patients ages 12 and older, the company announced. The recommended regimen is for Jivi to be administered intravenously (into the blood) two times a week (30-40 IU/kg),…
uniQure has treated the first patient in a Phase 2b dose-confirmation study of its investigational gene therapy, AMT-061, for people with severe and moderately severe hemophilia B. The trial is still recruiting participants. AMT-061 uses a viral vector, AAV5, to deliver the gene for a mutated clotting factor…
The Royal College of Surgeons in Ireland (RCSI) and Bayer will collaborate to develop improved treatments for patients with severe hemophilia. The research collaboration will focus on personalized treatments adapted to the severity of each patient’s condition to achieve safer and more effective blood clotting in hemophilia…
Catalyst Biosciences announced positive interim results from a Phase 2/3 clinical trial (NCT03407651) of the Factor VIIa variant marzeptacog alfa (activated) (MarzAA) in the preventative treatment of patients with hemophilia A or B with inhibitors. The study, aiming to include up to 12 patients, is recruiting in Russia…
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