José Lopes, PhD, managing science editor —

A gene therapy from uniQure in early testing, called AMT-180, has the the potential to treat all hemophilia A patients, including those with inhibitors, according to the company. Hemophilia A is caused by missing or defective factor VIII (FVIII), a clotting protein. About 30% of patients with severe hemophilia A develop inhibitors, or…

uniQure is continuing to enroll participants for its global Phase 3 trial (NCT03569891) testing the potential gene therapy AMT-061 in men with severe or moderately severe hemophilia B, according to an update from the company. In addition, uniQure announced that three patients included in a Phase 2b trial (…

Total joint replacement (TJR) is more common in severe hemophilia patients requiring more frequent treatment, according to a population-based study in Taiwan. The findings also revealed that the incidence of TJR does not differ between hemophilia A and B. The study, “Comparison of Total Joint…

Hemophilia patients may now benefit from financial assistance for out-of-pocket expenses, health insurance premiums, and incidental medical costs following the launch of a new program from The Assistance Fund (TAF). Founded in 2009, TAF is an independent nonprofit aimed at helping eligible patients and their families cover costs not…

Oxidation of clotting protein factor VIII (FVIII) increased the immune response to the replacement therapy in a mouse model of severe hemophilia A, a study found. Conducted by researchers in France, the study, “Oxidation of factor VIII increases its immunogenicity in mice with severe hemophilia…

Hemophilia patients who develop inhibitors against treatment face markedly higher healthcare costs — about three times those of other patients — and experience poorer quality of life, according to a team of scientists in Italy. Alternatives are available and should be considered for these patients, they write. The study, titled…

Preliminary results of a Phase 1/2a trial of the hemophilia A experimental treatment BIVV001 show a significant extension of replacement therapy lifetime in the blood, Bioverativ announced. The ongoing, and still recruiting, open-label, multicenter EXTEN-A study (NCT03205163) is evaluating the safety, tolerability, and pharmacokinetics of a single intravenous injection…

Chronic joint inflammation lowers the health-related quality of life in people with severe hemophilia, a European study reports, and urges physicians to be aware of this association in treating patients. The research, “The impact of severe haemophilia and the presence of target joints on health-related quality-of-life,” appeared in…

Researchers are creating a free web-based calculator that will help physicians determine the best patient-specific preventive regimens for children with hemophilia, according to a recent study. The tool is based on newly developed methods that identified key features of an effective preventive regimen. This includes giving Factor VIII (FVIII) injections…

Many hemophilia B patients experience joint pain, depression and overall poor health, a study reports. Other findings were that women with hemophilia B had worse health than men and that those caring for children with the disorder often experience anxiety and depression. The team titled its study in the European Journal…