Lindsey Shapiro, PhD,  science writer—

People with moderate-to-severe hemophilia and ankle joint disease reported significant impairments in life quality and foot and ankle function, according to a recent U.K. study. Chronic pain emerged as a significant predictor of these patient-reported outcomes, as did the presence of inhibitors — neutralizing antibodies that can lower the…

Note: This story was updated Feb. 24, 2023, to correct the secondary headline since there are several other recombinant factor VIII therapies available.  Altuviiio is a first-in-class therapy, but not the first. Efanesoctocog alfa, Sobi and Sanofi’s first-in-class, long-lasting recombinant or man-made factor…

Hemophilia A patients at a single center in France reported high rates of satisfaction with Hemlibra (emicizumab) after a year of real-world use, a study has found. Patients reported improvements in health and life quality, as well as reductions in pain and bleeding with the therapy, but problems…

Researchers have identified immune system proteins that might drive the development of inhibitors, or neutralizing antibodies, against factor VIII (FVIII) replacement therapies in people with hemophilia A. These proteins, called complement proteins, appear to work collaboratively with danger signals that alert the body to critical situations in order to…

High levels of antibodies against clotting factor VIII (FVIII) were associated with a slower response to immunosuppressive treatments in a small group of people with acquired hemophilia A in new study in China. Of 34 patients, 29 achieved a complete response to their first-line immunosuppressive therapy. Still, a lower…

An investigational factor VIIa (FVIIa) therapy called GEN-0828 may be able to minimize blood loss and accelerate blood clotting at lower doses than NovoSeven, an approved medication used to control bleeding in hemophilia patients, according to a study in a mouse model of hemophilia B. Still, both treatments…

The positive safety and efficacy profile of Hemgenix (etranacogene dezaparvovec) — the first and only approved gene therapy for adults with hemophilia B — was sustained for two years, according to data from a Phase 3 trial, CSL Behring has announced. Findings were generally similar to previous…

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…

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…

Adults with moderate-to-severe hemophilia engage in less physical activity and have reduced physical fitness relative to healthy adults, according to a recent study. Data also indicated these patients had more indicators of abdominal fat accumulation and more often had high blood pressure than healthy people, which could overall reflect…