The cost of the one-time hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) will now be covered for eligible patients in Germany through the country’s public healthcare system. This comes through a reimbursement price agreement between developer CSL Behring and GKV-Spitzenverband, the German National Association of Statutory Health…
Despite high rates of bone health issues in hemophilia and von Willebrand disease (VWD), the routine use of DEXA scans remains low across hemophilia treatment centers (HTCs) in the U.S., a study reports. DEXA, or dual-energy X-ray absorptiometry, is a type of imaging test that uses low-dose X-rays to…
The U.S. Food and Drug Administration (FDA) has approved fitusiran as a routine prophylactic therapy to prevent or reduce the frequency of bleeding episodes in people with hemophilia A or B, ages 12 and older, with or without inhibitors, or antibodies that can make treatment less effective.
Prophylactic, or preventive, treatment with Alhemo (concizumab) is associated with better quality of life and a lower treatment burden for people with hemophilia A or B without inhibitors, according to patient-reported data from a Phase 3 trial. The ongoing study, called explorer8 (NCT04082429), is investigating the…
About 30% of people hospitalized due to acquired hemophilia have a bleeding event, while 17% report a thrombotic, or blood clot-related complication, such as stroke and deep vein thrombosis, a nationwide U.S. study finds. Patients ages 65 years and older, and those with thrombotic events were more likely to…
Takeda Pharmaceuticals has decided to discontinue Hemofil M (human antihemophilic factor) and Recombinate (recombinant antihemophilic factor), both of which are approved as replacement therapies to prevent and control bleeding episodes in children and adults with hemophilia A. “This was not a decision we made lightly,” Anthea Cherednichenko,…
Children with hemophilia have problems with dynamic balance — the ability to maintain balance while moving — and an increased risk of falls compared with healthy peers, according to a new study by two researchers in Turkey. The findings showed, however, that such impairments were not related to joint…
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…
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…
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