Healthcare costs, particularly related to disease-specific medications, are especially high for people with hemophilia A or hemophilia B on preventive treatments and/or for those with inhibitors. That’s according to a study in France, which also found that patients have a significant clinical burden, with a higher risk of…

The U.K.’s National Institute for Health and Care Excellence (NICE) has recommended Altuvoct (efanesoctocog alfa) be available through the National Health Service (NHS), England’s public healthcare system, as a treatment option to manage and prevent bleeds in people with severe hemophilia A, ages 2 and older. This decision…

Second in a series. Read part one. A hemophilia inhibitor is like a firewall in a computer network. Clotting factor replacement therapy is supposed to enter the bloodstream and help form clots to stop bleeding, just as data should pass through a network without interruption. However, an inhibitor acts…

Ixinity (trenonacog alfa) can effectively prevent and control bleeds in young children with severe or moderately severe hemophilia B, according to data from a Phase 3/4 clinical trial. “This study showed that [Ixinity] was safe and effective as prophylaxis and for controlling bleeds in previously treated persons with…

The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has approved efanesoctocog alfa — to be sold under the brand name Altuvoct —  as a treatment for moderate or severe hemophilia A in adults and children, ages 2 and older. Approved in the European Union in June…

Women like me often wonder how the hemophilia community has overlooked us for so long. The stories of women labeled as mere “carriers” — despite experiencing life-threatening bleeding episodes — are endless. For decades, we’ve lived with symptoms that disrupt our lives, yet we were dismissed, misdiagnosed, or told…

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…

Small vesicles in the saliva of people with severe hemophilia A may explain why these patients rarely have bleeds in their mouth and throat. The vesicles contain a protein complex that’s able to induce blood coagulation in these patients, thereby reducing bleeding. The findings may help “develop innovative approaches…

For the first time in Denmark, people with hemophilia B have been treated with the gene therapy Hemgenix (etranacogene dezaparvovec), according to an announcement from CSL Behring, the company that markets the therapy. Hemgenix was cleared for use in the European Union in early 2023, following its…

Be Biopharma has raised $92 million in series C financing to support the first-in-human Phase 1/2 trial of BE-101, the company’s B-cell therapy candidate for hemophilia B. Proceeds from the funding round will be partly used to generate proof-of concept data from the BeCoMe-9 trial (NCT06611436), which…