News

Dimension Therapeutics’ gene therapy candidate drug DTX101 shows promising results by improving the levels of blood clotting factor IX (FIX) in adult patients with moderate/severe to severe hemophilia B, the company said. Hemophilia B is caused by FIX deficiency, due to mutations on the gene that encodes this protein. Treating…

Although gene therapy for hemophilia has reached clinical testing in humans, researchers continue to work toward improved delivery systems of the treatment — not an easy task considering the large size of the human factor VIII gene. In a study titled “Characterization of Adeno-Associated Viral Vector-Mediated Human Factor…

CSL Behring, a biotherapeutics firm based in King of Prussia, Pa., has awarded Local Empowerment for Advocacy Development (LEAD) grants of $10,000 each to four U.S. nonprofit groups that advocate for patients with hemophilia and other bleeding disorders. The funds will help these organizations make their voices heard on Capitol Hill…

South Korea has approved an Investigational New Drug (IND) application for Catalyst Biosciences’ experimental therapy CB 2679d/ISU304, a highly potent next-generation coagulation Factor IX variant for the treatment of hemophilia B. Catalyst focuses on developing medications for hematology conditions (blood disorders), which include coagulation factors that can prevent spontaneous…

Novo Nordisk’s Refixia (nonacog beta pegol, N9-GP) received a positive opinion by the Committee for Medicinal Products for Human Use (CHMP) for treatment of adolescents and adults with hemophilia B. The CHMP, under the European Medicines Agency (EMA), recommends the granting of a marketing authorization in Europe for…

An infant was diagnosed with severe hemophilia A after developing splenic injury (injury to the spleen), a rare condition among newborns. For the first time, however, a newborn with these conditions was successfully treated with recombinant factor VIII replacement therapy without the need for surgical intervention, doctors reported. The report, titled…

Hemophilia B hurts educational and job prospects for both adults with the disease and caregivers of hemophiliac children, a recent study found. The study, “Impact of mild to severe hemophilia on education and work by US men, women, and caregivers of children with hemophilia B: The Bridging…

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…

NovoSeven stops or slows bleeding in children with hemophilia but is associated with blood clotting, a study of hospital medical records indicates. The team that did the retrospective study said more risk-benefit studies of children’s use of NovoSeven are needed. NovoSeven is also known as recombinant activated factor VII – rFVIIa. The…

Gene therapy delivered by microbubbles shows promise in treating hemophilia B, a hereditary bleeding disorder caused by the lack of blood clotting factor IX, new research suggests. The study, “Ultrasound-targeted hepatic delivery of factor IX in hemophiliac mice,” appeared in the journal Gene Therapy. Hemophilia B affects approximately…