Hympavzi (marstacimab-hncq) may be available by prescription in the U.S. before the end of the year. The therapy was approved this month as a routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages 12 and older, with hemophilia A or…

People with hemophilia who report better mental health may be more likely to follow their prophylactic (preventive) treatment as prescribed, according to a new study based on a survey of 78 adolescent and adult males in Brazil. Researchers said this finding “underscores the need for [clinicians treating people with…

A Phase 4 trial investigating Ixinity as a prophylactic treatment for children under age 12 with hemophilia B has reached the halfway mark for its enrollment target, Medexus Pharmaceuticals announced. The international study (NCT03855280), taking place at sites in Brazil, Colombia, Moldova, South Africa, and Ukraine, continues recruiting eligible…

Living with hemophilia involves more than managing physical symptoms. It comes with mental and emotional challenges, too. My husband, Jared, who has severe hemophilia B and epilepsy, has spent much of his life feeling the need to prove himself. From childhood to adulthood, this drive for validation shaped…

Preventive treatment with Eloctate (efmoroctocog alfa) for up to five years led to sustained reductions in all bleed types for children, adolescents, and adults with severe hemophilia A, according to data from two Phase 3 trials and their open-label extension study. New analyses of these pivotal trials also…

When my oldest son was diagnosed with severe hemophilia at birth, I found myself catapulted into a world I never knew existed. I became part of the bleeding disorder community, like it or not. I slowly began to realize that this community was filled with passionate, well-educated people who knew…

“Be prepared.” Scouts around the world have this motto ingrained in their hearts. While I was a Girl Scout for only a short time, this practice remains essential to me. I look ahead and consider the numerous ways a situation may manifest. As the mother of two sons with…

Some days it’s easy to see the cracks in our family’s situation — the unpredictable costs, the logistics of keeping medication on hand, the mental load of being a partner to someone with severe hemophilia B and epilepsy. But every so often, I remind myself that there are…

A single infusion of AMT-061 (etranacogene dezaparvovec), uniQure’s experimental gene therapy for the treatment of hemophilia B, led to increased and sustained activity of clotting factor IX (FIX), according to one-year data from a Phase 2b clinical trial. The therapy effectively prevented…

For many years, those who suffered from hemophilia were told to not take part in any kind of physical exercise. It was only in the last 40 years or so that doctors started encouraging patients to choose a physical activity that’s both safe and enjoyable for each individual. Play safe…