Researchers have found that the high price of recombinant clotting factors for patients with hemophilia B is the main cause of the economic burden of the disease in the United States. However, despite the cost, prophylaxis treatment can provide clinical benefits to patients that may reduce other costs not…

A hemophilia gene therapy developed by Spark Therapeutics and Pfizer nearly eliminated bleeding episodes in 10 patients who took part in a Phase 1/2 trial. The treatment, SPK-9001, worked so well in eight of the 10 that they were able to stop taking clotting factor replacements. Researchers published the preliminary trial…

Hemgenix (etranacogene dezaparvovec), the first approved hemophilia B gene therapy, has been successfully administered to two patients in France, marking the first time the treatment has been given in a real-world setting in Europe. The gene therapy, originally developed by uniQure and marketed by CSL Behring, was…

Many boys and young men with hemophilia regularly participate in sports. However, doing so frequently — particularly three or more times a week — may raise the risk of sports-related bleeding, especially during early adolescence, a Japanese study reports. “Regardless of sports type, most bleeding episodes happened during junior…

Last in a series. Read parts one and two. In the previous columns of this series, hemophilia awareness advocate Lee Hall shared his memories of a childhood shaped by hospitalizations and the painful reality of early hemophilia treatment, followed by the devastating era from the 1970s…

Treatment with the investigational gene therapy Roctavian (valoctocogene roxaparvovec) can reduce bleeding frequency and the use of replacement therapies, while maintaining or improving the quality of life of people with severe hemophilia A for up to five years, updated Phase 1/2 trial data show.  “These…

My mighty warrior Caeleb is about to start a new chapter in his life with hemophilia; it’s one I never imagined I’d see. About nine months ago, Caeleb began a new treatment with a medication that has been taking the bleeding disorder world by storm. Hemlibra (emicizumab-kxwh)…

Mobility problems, bleeding, pain, and uncertainly in daily life are the biggest challenges people with hemophilia are facing, according to a survey from six central European countries. Additionally, while most respondents, including caregivers, felt well informed on hemophilia, they continue to seek further education on the condition and new…

Hemophilia has a substantial impact on sexual function and well-being for many men with the disease, but they rarely discuss it with their doctors, a study from the Netherlands found. “To address this gap, tools designed to provide and assess information about sexual functioning and well-being to normalize the…

When I was diagnosed with hemophilia B and von Willebrand disease as a child, there were no choices in terms of treatment strategy. Treatment for me came in the form of intravenous blood products administered only in the hospital. Fast-forward to today, and I’m fortunate to have options…