Marisa Wexler, MS,  senior science writer—

A single injection of uniQure‘s gene therapy AMT-061 (etranacogene dezaparvovec) is well-tolerated, effectively increases the levels of clotting factor IX (FIX), and completely prevents bleedings without the need for additional FIX injections in people with hemophilia B, even in the presence of neutralizing antibodies against the treatment,…

The Fitbit wristband can help with the continuous monitoring of physical activity in people with hemophilia, but other strategies are needed to encourage increased activity, a study suggests. The study, “Physical Activity Monitoring and Acceptance of a Commercial Activity Tracker in Adult Patients with Haemophilia…

The gene therapies AMT-060 and AMT-061 both restored coagulation factor IX levels in a primate model of hemophilia B, but AMT-061 resulted in greater coagulation activity at the same dose, a new study reports. The study, “Enhanced Factor IX Activity Following Administration of AAV5-R338L “Padua” Factor IX…

Problematic thrombotic events are fairly rare in people using NovoSeven, among all types of bleeding disorders for which it is approved, including hemophilia, a review study shows. The study, “Thrombotic events with recombinant activated factor VII (rFVIIa) in approved indications are rare and associated with older age,…

New data from clinical studies suggests that long-term preventive treatment of severe hemophilia A with Eloctate, or severe hemophilia B with Alprolix, increased quality of life and reduced annual bleeding rates. The data was presented in Melbourne, Australia at ISTH 2019, the 27th Congress of the International Society…

Health Canada has approved Esperoct, an engineered formulation of clotting factor VIII, for the treatment of hemophilia A in people of all ages. The approval covers prophylactic, or preventive treatment, on-demand use for controlling bleeding episodes, and use in reducing bleeding during surgery. Esperoct received similar approval…

Health Canada has approved Hemlibra (emicizumab) for people with Hemophilia A without factor VIII inhibitors. Hemlibra is an antibody therapy that works by combining factors IX and X of the blood clotting cascade, effectively “replacing” the function of factor VIII, which is lacking in Hemophilia A patients. It is…

Catalyst Biosciences announced the beginning of enrollment for a Phase 2b clinical trial for dalcinonacog alfa (DalcA) in people with Hemophilia B. Hemophilia B is caused by the lack of a functional Factor IX clotting protein. Like many available therapeutics for Hemophila B, DalcA is essentially a lab-manufactured version…