Combining immune tolerance induction, used to prevent the development of anti-factor VIII inhibitors, with Hemlibra (emicizumab) is a feasible and safe way of treating children with severe hemophilia A, a study shows. Published in the journal Haemophilia, the report covers clinical outcomes in the first seven patients to be…
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Hemophilia A patients undergoing total hip or knee replacement/reconstruction surgeries have lesser blood loss and require a lower transfusion amount if treated with Cyklokapron (tranexamic acid), a study reports. They also are likely to experience less joint pain or swelling and better joint function, and have lower levels…
While there is no cure for hemophilia, it is very possible to live with this genetic bleeding disorder and maintain a good quality of life. Proper nutrition and weight management combined with a safe and effective exercise plan monitored by a medical professional can make…
Switching to extended half-life (EHL) products has provided a reduction in the number of infusions and fewer bleeding events compared to standard treatments, particularly in hemophilia B, according to early real-world data from Europe. The study, “Real‐life experience in switching to new extended half‐life products…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Sigilon Therapeutics’ candidate cell therapy, called SIG-001, for hemophilia A. Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application…
Patrice Thomas has utilized her skills as a women’s healthcare advocate to learn about marginalized communities and support structural change. Through her current position at the Hemophilia Foundation of Michigan (HFM), Patrice is bringing together women with hemophilia and healthcare providers to educate and break down barriers…
Pfizer’s Expansion in North Carolina May Help Advance Manufacturing of Hemophilia Gene Therapies
Pfizer will invest an additional $500 million into the construction of its new manufacturing facility in Sanford, North Carolina, the company announced. According to the company, the facility could help advance the manufacturing of one-time gene therapies that use modified adeno-associated viral vectors, such as SB-525, which is…
Mild to moderate hemophilia B affects the relationships of adult patients, children with the disease and their caregivers in aspects such as telling others about the diagnosis, selecting a partner, and feeling bullied, according to an American study. The research, “Mild-severe hemophilia B impacts relationships of…
Although most patients report that discussing their sexual health with their doctors is helpful, hemophilia significantly affects the quality of their sex lives, according to the results of two studies. The research, “Hemophilia and sexual health: results from the HERO and B-HERO-S studies,” was published in…
Preventive treatment with Hemlibra (emicizumab) is superior to replacement therapy with factor VIII infusions for reducing bleed rates in people with hemophilia A without inhibitors, a pooled analysis of trials shows. The study “Efficacy of emicizumab prophylaxis versus factor VIII prophylaxis for treatment of hemophilia A…