News

Therapy to prevent bleeding (called prophylaxis) in patients with severe hemophilia A led to decreased joint bleeding when it was started at any age. However, improved joint motion was only effective if prophylaxis was started before age 4 in non-obese patients, according to researchers. The study, “Prophylaxis Usage, Bleeding…

Men with hemophilia who have a Medicaid health plan spend less on healthcare than those with employer-sponsored health insurance, due to lower reimbursement. However, Medicaid spends more on extensive healthcare users than employer-sponsored plans, according to a new study from the Centers for Disease Control and Prevention (CDC). The study,…

A new, modified version of recombinant factor VIII (rFVIII), called rVIII-SingleChain, showed great efficacy and safety in treating bleeding events in children with severe hemophilia A. The study, “Safety, efficacy and pharmacokinetics of rVIII-SingleChain in children with severe hemophilia A: results of a multicenter clinical trial,” was published in…

Being unemployed or under-educated can have a negative effect on hemophilia patients’ quality of life, including their pain levels and ability to function, according to the results of a questionnaire that were presented at the ASH Annual Meeting and Exposition. In the patients surveyed, increased pain, functional impairment and reduced…

BioMarin Pharmaceuticals is moving forward with BMN 270, an experimental gene therapy for the treatment of hemophilia A, and is preparing new clinical trials expected to begin this year. The European Medicines Agency (EMA)’s Committee for Advanced Therapies (CAT) and its Committee for Medicinal Products for Human Use (CHMP) agreed that BMN 270…

The U.S. Food and Drug Administration (FDA) has granted uniQure’s AMT-060, an investigational gene therapy, breakthrough therapy designation for patients with severe hemophilia B. The regulatory agency based its decision on data from the ongoing, dose-ranging Phase 1/2 clinical trial (NCT02396342) evaluating AMT-060 for hemophilia B. The study’s estimated completion date…

Catalyst Biosciences will present three posters with data from its two investigational candidates for hemophilia at the upcoming 10th Annual Congress of the European Association of Haemophilia and Allied Disorders (EAHAD) Feb. 1-3 in Paris, France. The first poster presentation is for a next-generation factor VIIa, marzeptacog alfa (activated), that showed…

People with hemophilia may benefit from mild exercise to minimize their risk of bleeding according to a study published in the scientific journal Haemophilia.

A daily infusion of low-dose factor VIII (FVIII) improves trough levels in patients with severe hemophilia A — compared to infusions performed every other day — without increasing factor VIII consumption, or the amount of medication a patient must take, researchers have found. Trough levels are the lowest concentrations of a…

The hemophilia A treatment Nuwiq (simoctocog alfa) is safe and effective for patients with severe disease, both when used on demand or as a preventative treatment, researchers determined in a recent report. The researchers, who analyzed available data on Nuwuq, detailed their finding in the article, “Efficacy and…