When someone has a hemophilia diagnosis, they receive treatment in one of two ways: on demand, in which they are given products to help their blood clot after an injury or before a scheduled medical procedure; or prophylaxis, which…

Bayer’s Jivi, an FVIII replacement therapy for hemophilia A, has a longer half-life and a slower clearance from blood circulation than Eloctate, and may protect against bleeding for longer periods, results of a Phase 1 trial in people with severe disease suggests. The study “Direct comparison of…

Based on feedback from the hemophilia B community, Aptevo Therapeutics announced that a 3,000 international unit (IU) vial assay of Ixinity  is now available for patients preferring the convenience of a larger assay over the smaller IUs on the market for this intravenous recombinant factor IX. The…

The ongoing Phase 1/2 Alta trial continues to show that a single infusion of SB-525, an investigational gene therapy, leads to stable and clinically relevant improvements in clotting factor VIII and marked reductions in the need for replacement therapy in patients with hemophilia A. This preliminary follow-up data…

In the past, no one believed women could have hemophilia. The medical community assumed that one X chromosome would compensate for the other. Doctors now know about the process of duplicate X chromosome inactivation, called “lyonization.” As the inactivation process is random, some female…

Self-reported health outcomes of hemophilia patients provide useful and clinically valuable data on the efficacy of treatments, a population-based study shows. The study, “Real-world utilities and health-related quality-of-life data in hemophilia patients in France and the United Kingdom,” was published in…

For the second year, key pharmaceutical and academic leaders in hemophilia therapy development will gather at the Hemophilia Drug Development Summit — to be held in Boston Aug. 20–22 —to discuss and advance the next generation of safe and more effective therapies for bleeding disorders. The recent approval of  Genentech’s …

Most people will tell you that their children are opposites. One is outgoing, the other would rather be left alone. One will talk until your head hurts, the other will utter only a few words when prompted. Their personalities are different, yet they have the same biological parents. I see…

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…

It’s been nearly five months since I became a mom to baby Cittie. In those months, my life has been transformed into something completely different. My social media feed, which used to be full of items about academics and career, is now full of articles about babies and…