Hemophilia A patients at a single center in France reported high rates of satisfaction with Hemlibra (emicizumab) after a year of real-world use, a study has found. Patients reported improvements in health and life quality, as well as reductions in pain and bleeding with the therapy, but problems…
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Joint changes detected by magnetic resonance imaging (MRI), and often are missed on radiographs, are a strong predictor of damaging bleeds over the next five years in joints of people with hemophilia, a study suggests. A 10-fold increase in the five-year risk of joint bleeds was found by…
A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 safely and effectively prevented bleeding episodes and the use of clotting factor VIII (FVIII) for up to four years in men with hemophilia A, according to the latest data from a Phase 1/2 trial and its extension study.
Preventive treatment with marzeptacog alfa activated (MarzAA) can reduce bleed frequency substantially in men with hemophilia A or B who developed inhibitors to conventional replacement therapies, according to data from a Phase 2 clinical trial. The findings were reported in the study, “Subcutaneous engineered factor…
Preventive treatment with Esperoct (turoctocog alfa pegol) plus joint surgery significantly decreased annual joint bleeding rates, reduced pain, and improved mobility in hemophilia A patients, according to an analysis of Pathfinder trial data. “Our results therefore support the notion that patients with haemophilia A being treated with [Esperoct]…
Factor VIII (FVIII) levels in the bloodstream of men with severe hemophilia A treated with the investigational gene therapy Roctavian appear to be influenced by regulatory molecules involved in F8 gene activity and FVIII protein folding and release, a study reveals. Researchers also found a dose-dependent relationship between…
The European Medicines Agency (EMA) has accepted BioMarin Pharmaceutical’s request for accelerated assessment of Roctavian, the company’s investigational gene therapy for severe hemophilia A, for a second time. Last year, BioMarin’s regulatory applications for Roctavian (valoctocogene roxaparvovec) got rejected both in Europe and in the…
Elevated levels of the immune signaling protein BAFF were found in the bloodstream of boys who developed neutralizing antibodies against FVIII replacement therapy, a standard treatment in hemophilia A, a study reported for the first time. Changes in the gene that encodes the BAFF protein, involved in the growth…
With Christmas only a few weeks away, Hemophilia Association of the Philippines for Love and Service (HAPLOS) threw its annual Christmas party. It’s a modest event, but seeing people gather, spread cheer, and enjoy one another’s company is heartwarming. The party is also a time to express gratitude to…
A single dose of the experimental gene therapy Roctavian (valoctocogene roxaparvovec) prevents bleeds and the need for preventive treatment, or prophylaxis, for up to six years in men with severe hemophilia A, according to updated data from a Phase 1/2 trial. “With every year of observation in this…