News

The National Organization for Rare Disorders (NORD) is seeking participants for its survey-based study Living Rare, which aims to better understand the real-world lived experiences of people in the U.S. with rare diseases. Living Rare, the first large-scale study of its kind in the U.S., seeks to capture the…

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…

A greater range of motion before surgery is associated with better post-surgical outcomes for people with hemophilia who undergo knee replacement surgery. That’s according to a recent study that also found that having the surgery on both knees at the same time had a negative impact on the overall…

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…

March is Bleeding Disorders Awareness Month (BDAM), and patients, caregivers, advocates, and supporters are working together to call attention to and provide resources for coping with the bleeding disorders estimated to affect more than 3 million people in the U.S. Bleeding disorders such as hemophilia, von Willebrand disease, and…

Pfizer said it will stop development and commercialization of hemophilia B treatment Beqvez (fidanacogene elaparvovec-dzkt). “Pfizer has made the decision to cease further development and commercialization activities with respect to Beqvez for several reasons, including the limited interest patients and their doctors have demonstrated in hemophilia gene therapies…

The Loma Linda University Health (LLUH) Hemophilia Treatment Center has become the first clinic in California offering Hympavzi (marstacimab) for people with hemophilia B. “For the first time, patients with hemophilia B in Southern California have access to a therapy that is not just effective, but also drastically…

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…

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…