Preventive treatment with Hemlibra (emicizumab) sustainably improved physical health-related quality of life and led to fewer missed workdays among people with severe hemophilia A without inhibitors, an analysis of pooled data from two clinical trials found. According to researchers, these findings add to previous data showing that Hemlibra…

Sangamo Therapeutics has handed over the development of SB-525, a gene therapy for hemophilia A, to Pfizer, which will now advance the therapy into Phase 3 clinical trials. Pfizer has already started recruiting participants for the lead-in Phase 3 trial (NCT03587116). which will support…

Jim Christensen Jr., a hemophilia A patient who has supported the National Hemophilia Foundation (NHF) for more than three decades, has received the Lester A. Rosen Humanitarian and Achievement Award, given annually by Ameritas, for which he is an independent financial representative. With the award, the insurance company…

Despite the increasing use of preventive factor VIII (FVIII) replacement therapy, people living with hemophilia A in Japan still experience a significant disease burden, according to a recent study. Using information from health records databases, researchers found that while patients have used increasing doses and spent more on medical…

Clinical symptoms of acquired hemophilia A in elderly patients can vary widely, a series of six case reports reveals. Also, these patients should be managed individually in order to achieve the best care and outcome, researchers suggest. The cases were described in the study “Management of…

In real-world settings, Hemlibra (emicizumab-KXWH) can safely control bleeds in children with hemophilia A better than previous treatment regimens, a study reports. Hemlibra’s improved efficacy was more pronounced among patients with inhibitors, or neutralizing antibodies targeting factor VIII (FVIII), which is the clotting protein missing in people with…

Precision BioLogic has developed what it says is a better way of detecting levels of substances in hemophilia A patients’ blood that prevent clotting factor therapies from working as well as they should. It collaborated with Roche and Genentech in creating a kit it says can detect levels of the…

Researchers at Emory University School of Medicine have developed a small-scale model system to study bleeding and clotting of wounds. The miniature model also accurately reproduces the alterations observed in hemophilia A patients. Their work, “A microengineered vascularized bleeding model that integrates the principal components of hemostasis”, was published in…

Changes in the cellular microenvironment could influence immune responses against factor VIII (FVIII) replacement therapy in people with hemophilia A, a new study suggests. Data from the cell culture study showed that FVIII proteins that cause an immune reaction differ depending on which other molecules are present in…

In the final days of 2023, an adult with hemophilia A named Maxwell became the first person in the U.S. to receive treatment with the one-time gene therapy Roctavian (valoctocogene roxaparvovec-rvox) — approved in June of last year — outside of clinical trials. “Receiving this treatment is liberating…