In patients with hemophilia A or B who undergo dental extractions, the use of hemostatic treatments and factor prophylaxis is associated with decreased rates of bleeding, according to findings from a retrospective analysis published in the Journal of Clinical Medicine. Among patients with hemophilia, dental extractions are frequently associated with bleeding and the need for…
The US Food and Drug Administration (FDA) has extended its Biologics License Application review of valoctocogene roxaparvovec (Roctavian™), a novel gene therapy for individuals with severe hemophilia A, BioMarin Pharmaceutical announced. The announcement noted that the FDA determined that the 3-year data analysis from the phase 3 GENEr8-1 clinical trial represented a major amendment and…
Ankle arthrodesis performed in patients with hemophilic ankle arthropathy may improve pain and functioning while keeping revision and complication rates low, according to a study published in Haemophilia. The authors conducted a literature review to investigate the mid- to long-term postoperative outcomes of ankle joint fusion in patients with hemophilia. They sought to understand the…
A new study has found that recombinant factor IX-FIAV (FIX-FIAV) is effective in increasing thrombin formation and clotting activity in patients with hemophilia A, regardless of inhibitor status. The study, published in the Journal of Thrombosis and Haemostasis, confirms previous studies that showed FIX-FIAV improved the hemophilia A phenotype in vitro and in vivo. “Here…
Patients with severe hemophilia A should undergo regular bone mineral density (BMD) screenings, according to findings from a study conducted in Bulgaria and published in the journal Folia Medica (Plovdiv). Individuals with severe hemophilia experience spontaneous bleeding into their weight-bearing joints, which is linked with mutations in the factor VIII (FVIII) gene that lead to…
An easy-to-use, updated, interactive database of 1692 variants in coagulation factor IX (FIX) offers detailed insight into hemophilia B, according to findings from a review published in the Journal of Thrombosis and Haemostasis. Patients with hemophilia B exhibit deficiencies in coagulation FIX, which are associated with “genetic variants in the 33.5 kb F9 gene located…
UniQure Inc has announced positive results from the HOPE-B clinical trial on the efficacy and safety of etranacogene dezaparvovec-drlb (Hemgenix®), the first gene therapy for adults with hemophilia B. The results, announced via news release, have been published in the New England Journal of Medicine. “HOPE-B was . . . the first and only phase…
People with hemophilia A and B reported similar impairment to health-related quality of life (HRQoL), with their scores largely impacted by joint damage these patients experience regardless of prophylaxis, according to an article recently published in Haemophilia. The study enrolled 63 patients with severe hemophilia B and 63 matched controls with hemophilia A. All participants…
The US Food and Drug Administration has approved a new high-sustained factor VIII (FVIII) replacement therapy—efanesoctocog alfa (ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl])—for the treatment of hemophilia A. Efanesoctocog alfa is indicated for routine prophylaxis and control of bleeding episodes, as well as the perioperative management of adults and children with hemophilia. It is…
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Darlene Shelton, founder and president of Danny’s Dose Alliance, a Missouri-based nonprofit that seeks to ensure proper emergency medical care for all patients with hemophilia and other bleeding disorders. Read the full story here: Danny’s Dose Alliance Fights for Proper Emergency Care for Those With Bleeding…
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