A single infusion of Hemgenix (etranacogene dezaparvovec-drlb) helped boost levels of factor IX (FIX), the clotting protein missing in hemophilia B, in many patients who already had antibodies against the viral carrier, according to new trial data. The findings suggest the gene therapy may benefit a broader group of…
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The sound of a tape gun gives me anxiety. There’s the quick pull of the tape from the roll, followed by its crinkling as it’s pressed onto the box’s flaps. The serrated blade bites down and tears the tape cleanly, the cardboard often giving a faint, hollow thud beneath the…
Healthcare costs of a hospitalization for children and adults with hemophilia in the U.S. are higher than for those who do not have a bleeding disorder, a study reported. Hospitalizations for children with hemophilia were also seen to be mostly driven by bleeding and catheter-related infections, while high blood pressure and other…
Note: This story was updated Jan. 20, 2026, to correct data on the number of boys on preventive FIX replacement therapy at the time of the analysis. Using longer-acting factor IX (FIX) products — known as extended half-life (EHL) products, or EHL-FIX — safely reduced factor use and helped children…
Preventive treatment with Hemlibra (emicizumab) sustainably improved physical health-related quality of life and led to fewer missed workdays among people with severe hemophilia A without inhibitors, an analysis of pooled data from two clinical trials found. According to researchers, these findings add to previous data showing that Hemlibra…
A new online service allows patients with bleeding disorders like hemophilia to share important information about their treatment and bleeds with their medical team in real time. MORE: Five tips for living healthily with hemophilia The HemaGo XChange portal was created by Novo Nordisk A/S as an update to their existing website and…
A new online service allows patients with bleeding disorders like hemophilia to share important information about their treatment and bleeds with their medical team in real time. MORE: Five tips for living healthily with hemophilia The HemaGo XChange portal was created by Novo Nordisk A/S as an update to their existing website…
I’m 25 weeks pregnant. Now that I’ve passed the halfway point of my pregnancy, I’m full of thoughts about the future. What happens when I give birth? After I give birth? When it sinks in that we are parents? Based on my ultrasound scans, it looks like we will have…
Marstacimab, an experimental treatment in clinical testing for hemophilia A and B, was able to restore clotting in hemophilic blood and plasma tested in laboratory assays, according to a study. If proven safe and effective in patients, the investigational therapy could provide an alternative preventive solution to…
Most young people with moderate or severe hemophilia A and B in France receive long-term prophylactic (preventive) treatment — an approach linked to a lower rate of bleeding episodes than among patients treated on demand. The study, “Choice of factor VIII/IX regimen in adolescents and young adults with…