Prescriptions for extended half-life products to hemophilia A patients in Japan increased from 2016 to 2020, according to a new study that analyzed real-world data from a hospital database. This was accompanied by an increase in healthcare costs seen for patients switching from standard half-life factor replacement therapies to…
Long-term prophylaxis or preventive treatment with Eloctate (efmoroctocog alfa) helps to ease pain and improve health-related life quality for adults and children with hemophilia A, according to an analysis of three Phase 3 clinical trials. “These analyses highlight the relevance of pain, physical health, and overall wellbeing as…
More than 1 in 10 men with hemophilia reported being restricted in their sexual functioning, which, in turn, had a negative impact on their quality of life, per data from a new survey-based study conducted in the Netherlands. The study also found that this…
A single dose of Roctavian (valoctocogene roxaparvovec-rvox) nearly zeroed out the number of bleeds and the need for prophylaxis, or preventive therapy, for as long as seven years — the longest follow-up to date for any hemophilia A gene therapy clinical trial. While most of the 12 patients…
The incidence of acquired hemophilia A (AHA) was nearly twice as high in Hong Kong than in Caucasian populations previously observed, a study found. Patients in Hong Kong tended to be older and have co-existing medical conditions, with age being a significant predictor of mortality and failure to achieve…
Brazilian hemophilia A patients who are 19 or older had significantly higher median care costs per patient per year than younger patients, a study reported. The study also revealed an increase in median costs for patients with more severe disease — that is, those who have less than 1%…
In an effort to cut costs and boost profitability, Biomarin Pharmaceutical is limiting commercial development of hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec-rvox) to three countries: the U.S., Germany, and Italy. The company has struggled to turn a profit from the gene therapy, which was approved in…
Prophylactic treatment for hemophilia can help people with the disease avoid unemployment due to disability, a study reports. Hemophilia has a negative effect on a person’s employability. A 2022 global report by the World Federation of Hemophilia indicates about 18% of hemophilia patients see their employment status impacted…
A 39-year-old man from Arizona became the first patient in Nevada to receive Hemgenix (etranacogene dezaparvovec), the first gene therapy to be approved for hemophilia B. The infusion was administered at the Cure 4 The Kids Foundation, under the supervision of Aimee Foord, director of the foundation’s bleeding…
Durveqtix (fidanacogene elaparvovec), a gene therapy for adults with severe and moderately severe hemophilia B, has been granted conditional marketing authorization by regulators in the European Union. The decision by the European Commission follows a positive recommendation on the …
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