The lives of four inspiring patients with bleeding disorders were introduced at the “Hope Ignites: Stories to Inspire” patient program at the Hemophilia Federation of America (HFA) annual symposium recently in Providence, Rhode Island. Sponsored by Octapharma USA, the “Hope Ignites” symposium was for patients with hemophilia to offer hope…
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All of the hemophilia B patients in an ongoing Phase 1/2 clinical trial of SPK-9001 experienced clotting-factor increases and reductions in bleeding episodes, according to the therapy’s maker, Spark Therapeutics. University of Pennsylvania medical researcher Adam Cuker will present the updated preliminary results today at the Hemostasis and Thrombosis Research Society (HTRS) 2017…
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…
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CSL Behring Awards Grants to 4 Nonprofits Representing Hemophiliacs, Other Rare Disease Patients
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…
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Hemophilia B Hurts Education, Job Prospects for Both Adult Patients and Caregivers, Study Finds
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…
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