News

The hemophilia B treatment Rebinyn is available in the United States, its maker Novo Nordisk reports. Rebinyn, which is administered intravenously, replaces the blood clotting factor IX that is missing in the disease. The U.S. Food and Drug Administration approved it in May 2017 to control bleeding episodes and manage bleeding…

Chris Bombardier has just made history by becoming the first person with hemophilia ever to scale the seven summits this January. Bombardier climbed each continent’s highest peak to raise awareness for hemophilia — and to show that with adequate training, medical assistance, preparation and willpower, even someone with a bleeding…

Catalyst Biosciences recently announced that top-line data from the Phase 1/2 trial of CB 2679d/ISU304 in patients with hemophilia B will be presented at the 11th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), Feb. 7-9 in Madrid, Spain. The oral presentation, titled…

Clinical trials of Hemlibra (emicizumab-kxwh) continue to show significant reductions in bleeding episodes in patients with hemophilia A with inhibitors, Genentech recently announced. Data from Hemlibra’s clinical development program were presented at the 59th American Society of Hematology (ASH) Annual Meeting. The results show that in hemophilia A patients with inhibitors…

This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 million Americans. The campaign kicks off Feb. 1 and culminates with Rare Disease Day on Feb. 28. Organized by…

Europe’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion supporting the approval of Hemlibra (emicizumab) for routine prophylaxis of bleeding episodes in hemophilia A patients with factor VIII inhibitors. Hemlibra is a humanized monoclonal antibody that binds to activated factor IX and factor X to…

New research shows that bilateral total knee arthroplasty — or reconstruction of the joint — is a safe and cost-effective treatment for patients with hemophilic arthropathy (a hemophilia degenerative joint disease). The study, titled “Simultaneous bilateral total knee arthroplasty in patients…

A major complication in scientists’ efforts to treat hemophilia A by replacing a missing or defective clotting factor is an immune response to the treatment. Researchers have discovered a connection between the body’s production of pro-inflammatory molecules known as cytokines and its immune response to clotting factor VIII therapy. They used…

Researchers report that hemophilia interferes with patients’ sexual activity and is linked to worse health status. The study titled “Sexual Health in Patients with Hemophilia; The Insights from the Patient Reported Outcomes, Burdens and Experiences (PROBE) Study,” appeared in the journal Blood. Previous results from a small…

Researchers have found  that N8-GP (turoctocog alfa pegol) treatment improved the quality of life and satisfaction of children with severe hemophilia A. The study titled “Health-Related Quality of Life and Satisfaction of Children with Severe Hemophilia a Treated with Turoctocog Alfa Pegol (N8-GP),” appeared in the journal Blood.