Spontaneous and joint bleeds, and limits on physical activity are evident in people with severe hemophilia A even when they adhere to and remain on preventive treatment, an analysis of global, real-world data showed. These findings highlight the need for additional alternative therapies for patients with severe disease. The study,…

Tiny particles containing RNA led to successful delivery and production of factor VIII (FVIII) — the clotting protein that is missing or defective in people with hemophilia A — in a mouse model of the disease, according to a new study. The study, “Functionalized lipid-like…

Monthly treatment with the investigational medication fitusiran can reduce bleeding in people with hemophilia A and B, with or without inhibitors, according to data from two Phase 3 clinical trials. “We are encouraged by the data from these initial Phase 3 studies demonstrating fitusiran’s potential as a new…

As time passes, I can feel both the joys and the pressures of fatherhood. My life has centered around our daughter’s needs and ensuring that she is always happy and smiling. Friends have instructed me on the importance of fatherhood. So many problems are caused by weak father figures. Often,…

A mechanism largely left unexplored was shown to help regulate factor VIII (FVIII) — the clotting protein that is missing or defective in people with hemophilia A — in a study in mice. While early and preclinical work, it is reported to be the first study of this mechanism…

With so many injuries lately, possibly because of a lack of exercise, I found it necessary to finally learn the ropes of self-infusion. Self-infusion of critical factor is important for me to prevent and treat bleeding episodes. As much as I have wanted to learn the craft, I…

After over a year without a gym membership, my wife, Cza, and I are back in our lifting den. We do it for fun and, of course, fitness. I’ve had several injuries during the past few months, all of which I can associate with the lack of physical exercise…

Switching to Hemlibra (emicizumab) can add more than $100,000 a year to healthcare costs for people with hemophilia A in the U.S. who were previously using preventive replacement therapies, according to a new analysis. “Increased use of [Hemlibra] may cause an increase in economic burden for payers without…

People with severe hemophilia A who switch from standard to extended half-life replacement therapies experience fewer bleeds and require less frequent infusions, an analysis of patients in Austria indicates. Whether these gains are reflected in lower overall treatment costs, however, is less clear, its researchers added. Data from this…

The U.S. Food and Drug Administration (FDA) has approved Shire’s Adynovate for the treatment of pediatric patients under 12 years of age with hemophilia A. Adynovate also was approved for use by both adults and children undergoing surgical procedures, to control bleeding episodes. Adynovate is a recombinant Factor…