News

1st Patient Dosed in Phase 3 Trial of Injectable MarzAA

The first participant has been dosed in a Phase 3 clinical trial testing marzeptacog alfa activated (MarzAA), an experimental under-the-skin therapy for hemophilia A and B patients with inhibitors. The study, called Crimson-1 (NCT04489537) and sponsored by the therapy’s developer, Catalyst Biosciences, is currently recruiting patients at…

Hemlibra Found Effective for Severe Hemophilia A in Clinical Setting

Preventive, or prophylactic, treatment with Hemlibra (emicizumab) safely and effectively managed bleeding in adults and children with severe hemophilia A, a real-world study has found. The study, “Emicizumab prophylaxis: Prospective longitudinal real‐world follow‐up and monitoring,” was published in the journal Haemophilia. Originally developed by Roche’s subsidiary…

US Healthcare Costs Average 25 Times Higher for Hemophilia B

Healthcare costs can be 25 times higher for hemophilia B patients than for individuals who do not have a bleeding disorder, according to a recent study. These findings reflect both direct costs of hemophilia treatment and a greater need for other healthcare resources, researchers noted. The study, “…

Scientists Target FVIII Inhibitor Formation in Hemophilia A

Scientists found the immune signaling protein BAFF can promote the formation of neutralizing antibodies, or inhibitors, against blood clotting factor VIII (FVIII), which can lower the effectiveness of FVIII replacement therapies used to treat people with hemophilia A.  They also discovered that hemophilia A…