The first Blood Advocacy Week, which promotes activities to showcase the importance of maintaining a safe and continuous blood supply and advocate for changes in policies that strengthen blood supply chains in the U.S., will take place April 24–28. Hemorrhage, or bleeding, is the leading cause of preventable death…
News
Concizumab has been approved as a preventive treatment in Canada for hemophilia B patients ages 12 and older who are positive for factor IX inhibitors and need a routine prophylactic to prevent bleeds or reduce their frequency. This is the first approval for the daily under-the-skin injectable therapy, which…
With an emphasis on patient advocacy, supporters of World Hemophilia Day will participate in a variety of activities on April 17 to heighten awareness of the disease and other bleeding disorders that are thought to affect 1 in 10,000 people globally. The World Federation of Hemophilia (WFH) organizes the…
Higher replacement factor doses and the use of preventive treatments are just two ways prescription practices for hemophilia have changed over time, according to a survey of specialists over 22 years. The surveys also show the impact of Hemlibra’s (emicizumab-KXWH) approval and showcase the expectations for gene therapy,…
About one-third of moderate-to-severely affected people with hemophilia perceived declines in their daily functional abilities over a period of three to five years, a Dutch study found. While no strong risk factors for such declines were identified, patients more likely to be affected were those who were obese, had…
Xoma closed a $9.6 million deal to buy from Aptevo Therapeutics the rights to receive a payment stream for sales of Ixinity (trenonacog alfa), an approved medication used to prevent and treat bleeding episodes in people with hemophilia B. Under the terms of the deal, Xoma will…
SerpinPC, an investigational treatment that Centessa Pharmaceuticals is developing for hemophilia, will advance to interventional Phase 2 clinical testing later this year. The studies will support Centessa’s registrational program for hemophilia B. Such a program involves clinical trials that ultimately will lead to the company seeking regulatory approval…
Despite preventive treatment, people with hemophilia A or B report lower quality of life, mainly due to poor joint health, a study in Sweden shows. No significant difference was found between these two patient groups, who report high frequency of pain, mobility problems, and mental health issues like anxiety…
Proteins from tardigrades — microscopic animals known for their ability to survive in extreme conditions — could be used to stabilize replacement therapies for hemophilia A, allowing treatments to be stored for longer periods of time without refrigeration, a proof-of-concept study shows. “Our work provides a proof of principle…
For children with severe hemophilia A who develop inhibitors — neutralizing antibodies that reduce the effectiveness of standard therapies — starting treatment to eliminate inhibitors earlier is associated with a higher chance of success, a recent study reports. The study, “Low-dose immune tolerance induction therapy in…
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