People with mild to moderate hemophilia, in particular women with the disease, face unique challenges, many that are specifically linked to a lack of information and insufficient engagement with other patients and healthcare providers, according to a panel of U.S. experts. In a recent study, these experts proposed new…
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In Brazil, hemophilia markedly impairs patients’ quality of life and ability to work, with up to 82.6% of people with the disorder being unemployed, a review study finds. Barriers to treatment include long distances to centers, lack of coordination of specialized and emergency care, and restricted access to rehabilitation.
As gene therapy is relatively new treatment option for hemophilia B, it's normal to have some questions about it. Here are answers to some commonly asked questions about gene therapy for hemophilia B.
Casebia’s two-stage gene therapy approach was successful in delivering the human gene coding for clotting factor VIII (FVIII) in a mouse model of hemophilia A, leading to stable and increasing levels of the factor, researchers said. “Our scientific team, led by Alan Brooks, Ph.D., have achieved a notable…
A single-center U.S. study found relatively little genetic testing was done for girls with a 50% risk of being a hemophilia carrier — people with the altered gene who don’t have hemophilia themselves but can pass it to their children — over a four-year period in Washington state.
Over the past few years, I have learned that bleeding disorders are complex, particularly for women. Hemophilia is not always easy to understand or treat. It can take women up to 16 years to receive an accurate diagnosis. Then treatment plans are created, which can be challenging…
A European Medicines Agency (EMA) committee has recommended the approval of Novo Nordisk’s concizumab as a prophylactic, or preventive, treatment for adults and children, ages 12 and older, with hemophilia A or B who have inhibitors. The recommendation, from the EMA’s Committee for Medicinal Products for Human Use…
Bemiltenase alfa, an experimental factor X (FX) activator, helped with on-demand bleeding control for people with hemophilia A and B who had developed inhibitors against standard factor replacement therapies, according to results from early clinical trials. Inhibitors are antibodies that may reduce the effectiveness of hemophilia treatments, making it…
Advate is an effective and well-tolerated treatment among patients with hemophilia A, according to an observational, long-term, Japanese study. The treatment showed better results when used as a preventive, rather than an on-demand, therapy. The findings were reported in an article titled “Inhibitor development, safety and efficacy of…
In a real-world study in South Korea, preventive treatment with Adynovate (rurioctocog alfa pegol) was shown to be effective at preventing bleeding episodes among most children and adults with hemophilia A — findings that line up with those from clinical testing. In fact, “76.1% of patients on prophylactic…