Author Archives: Alice Melão

FDA Approves Rebinyn for Hemophilia B Treatment

Novo Nordisk’s recombinant coagulation factor IX nonacog beta pegol (N9-GP), under the U.S. trade name Rebinyn, has been approved by the U.S. Food and Drug Administration (FDA) to treat adults and children with hemophilia B. The company expects to launch this new treatment in the United States early in 2018. The…

Researchers Review Guidelines on Primary Prophylaxis in Hemophiliac Children

Recent studies have addressed the importance of preventive, or prophylatic, treatment in severe cases of hemophilia, and its long-term benefits for children. The results may help establish guidelines and treatment recommendations leading to improved care for such kids. Both the World Health Organization and the World Federation of Hemophilia urge preventive treatment…

Hemophilia Patients End Up at Emergency Rooms Most Often for Hypertension and Injuries

Adult hemophilia patients use hospital emergency departments mainly due to cardiovascular events, while pediatric patients most frequently complain of injuries, according to researchers. U.S. emergency department (ED) visits by hemophilia patients had a national estimated cost of $60 million in 2012, the year studied. These observations resulted from the retrospective and…

Potential Hemophilia B Therapy Shows Promising Results in Mouse Study

A new delivery mechanism of mRNA therapy was found to efficiently reduce hemophilia B symptoms by correcting the protein deficiency that characterizes the disease in a mouse study. This potential new method might be a viable alternative for many clotting disorders. The study, “Systemic delivery of Factor IX messenger RNA…