News

Joint problems caused by chronic bleeding often trouble people with hemophilia, especially in the knees, ankles, and elbows. A paper by scientists in Spain and the U.S. reviewed existing literature on knee treatments for people with this disease, focusing on best current practices — including a preventive, if inconveniently lifelong, therapy — and future…

The genetic blood disorder hemophilia B, caused by factor IX (FIX) deficiency, can be effectively controlled with gene replacement therapies, particularly when used for prevention. But because barriers to prophylaxis that include intravenous use, frequent dosing and medical costs are many, new research for better therapies is emerging. Researchers at the University of Wisconsin School of…

San Rafael, California-based BioMarin Pharmaceutical will present interim data of an open-label, Phase 1/2 clinical trial of BMN 270, an investigational gene therapy treatment for hemophilia at the upcoming XXXII International Congress of the World Federation of Hemophilia (WFH) July 24-28 in Orlando, according to a news release. The late-breaking…

CSL Behring‘s new long-lasting recombinant factor VII single-chain therapy, Afstyla, for adults and children with hemophilia A, is now available nationwide. The drug, approved by the U.S. Food and Drug Administration (FDSA) in May, is the only single-chain product on the market specifically designed for reduction and control of…

Researchers investigated self-reported health-related quality of life (HrQoL) in children and adolescents with hereditary bleeding disorders, such as hemophilia A, and found no differences between patients and their healthy siblings and peers. The research paper, titled “Health-Related Quality of Life in Children and Adolescents with Hereditary Bleeding Disorders…

Researchers from the PharmedOut project at Georgetown University Medical Center analyzed how pharmaceuticals market directly to people with hemophilia and found that, while strategies are effective, further regulation and studies are necessary. The study, “Direct-to-consumer Marketing to People with Hemophilia,” was published in the PLOS Medicine Policy Forum.  PharmedOut involves doctors,…

Researchers, working with mice, have developed a new way of producing substantial numbers of gene-corrected liver cells with the possibility of repopulation in vivo. The strategy, which relies on the liver’s unusual regenerative skills, may hold promise for the treatment of diseases such as hemophilia B. The study by scientists at Oregon Health & Science University,…

Researchers at the University of Tokyo reviewed the status of physical activity among hemophilia patients to discuss strategies to encourage activity through a behavioral change approach by focusing on hemophilia-relevant factors such as benefits and bleeding risk, risk management of bleeding, physical activity characteristics, and difficulty in exercise adherence. The study,…

uniQure N.V., a leading pharmaceutical specialized in human gene therapy, has presented additional data from its clinical trial in hemophilia B patients at the 21st Congress of the European Hematology Association (EHA),  June 9-12, in Copenhagen. The Phase 1/2 clinical trial of AMT-060 titled “Trial of…

A recent clinical trial indicates that replacement therapy with plasma-derived factor VIII is associated with lower incidence for development of inhibitors in patients with severe hemophilia A. The study,“A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A,” was published in The New England Journal of…