News

Most people with hemophilia A on preventive treatment with Hemlibra (emicizumab) will experience spontaneous or traumatic bleeds at some point, according to a study using real-world data from Israel. Results also indicated the risk of spontaneous bleeds while on Hemlibra is higher for older individuals. “Our findings indicate that…

An online academic training program has been launched globally for people interested advocating for bleeding disorders care and treatment in their respective countries, as part of a partnership between the World Federation of Hemophilia (WFH) and the New York University (NYU) Robert F. Wagner…

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…

Preventive treatment with Hemlibra (emicizumab) safely and effectively reduced bleeding rates in children with hemophilia A who were treated at a center in Texas, according to a new report. “We demonstrate a successful experience with emicizumab prophylaxis and safe [surgical] approach with a focus on minimizing postoperative bleeding,”…

Using a baby’s own umbilical cells as vehicles to deliver factor VIII (FVIII) — the missing or defective clotting protein in hemophilia A — may be an effective, long-lasting, and more affordable therapeutic approach for this blood disorder, an early study suggests. The findings “lay the groundwork for future…

Nearly one-fifth of patients with mild hemophilia admitted to treatment centers in the U.S. are female, according to a large study of nearly 30,000 people. Yet, women and girls make up a smaller proportion of patients with severe or moderate hemophilia, ranging from less than 0.5% to a little…

Two pharmaceutical companies, LeaderMed Health Group and OPKO Health, are teaming up to develop, manufacture, and commercialize two experimental medications in China and other locations in Asia. One of the investigational medicines, Factor VIIa-CTP, is being developed as a potential treatment for hemophilia. “Through this joint venture, we…

The U.S. Food and Drug Administration (FDA) has restored its approval of BeneFIX as a routine prophylactic, or preventive, treatment for bleeds in children and adolescents under 16 with hemophilia B. Marketed by Pfizer, BeneFIX is a lab-made version of factor IX (FIX), the blood clotting protein that…

SerpinPC, an experimental hemophilia therapy, at high dose safely reduced overall bleed rates by up to 88% and spontaneous joint bleeds by up to 94% in adults with severe hemophilia A and B, who were not using preventive treatment, according to top-line results from a Phase 2a…

A report describes — for the first time, according to study authors — the case of a girl who has both hemophilia A and C. To date, only 10 such cases had been documented in medical literature, all occurring in men. The case report authors highlight the importance of…