News

The European Medicines Agency (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) has initiated a review of factor VIII-containing medicines, to evaluate the risk of inhibitor protein development in patients starting treatment for hemophilia A, according to a press release. This review, which will cover all medicines containing factor VIII authorized in the…

A recent study involving adolescent and young adult patients with hemophilia, to evaluate racial and ethnic disparities in following recommended clotting factor treatment regimens, chronic pain and quality of life, indicated that non-white patients report more chronic pain and worse quality of life. The research paper, “Racial Differences in Chronic…

A new study from Finland demonstrated that neutralizing antibodies (inhibitors) can develop in people with untreated hemophilia, and that early intensive prevention might prevent bleeding and inhibitor development. The research report, titled “Inhibitor development in previously untreated patients with severe haemophilia A: a nationwide multicentre study in Finland,” was published in…

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,…