UniQure‘s investigational gene therapy AMT-060 for severe hemophilia B has received PRIME designation by the European Medicines Agency (EMA). The EMA designation was based on the results of a Phase 1/2 open-label, dose-escalating study of AMT-060 (NCT02396342). The study included 10 patients, each receiving…

https://www.youtube.com/watch?v=0_EmvUb171I This video from Medic Tube gives an overview of the genetic causes behind hemophilia. The narrator explains that it’s very rare for females to have hemophilia because they need to inherit the faulty X gene from both of their parents, whereas males only need to inherit one…

Although hemophilia has no cure, there are ways to manage the disease and avoid bleeding episodes. We’ve put together a list of tips to help patients live well with hemophilia using information from the Mayo Clinic and the Centers for Disease Control and Prevention. MORE: World’s…

More than 12,300 hemophilia cases in developing countries were treated with therapy donations from Bioverativ and its partner Sobi in 2016, Bioverativ said in a press release about its participation in World Hemophilia Day. The company, which is promoting genetic testing in women and girls who could be susceptible to bleeding episodes, worked with other…

https://www.youtube.com/watch?v=IhVOXnB-0KQ In this video from Bleeding Disorders Community, Ryan shares his hemophilia story. He was diagnosed as a baby with severe hemophilia but he doesn’t let the disease stand in the way of his passion for fitness. MORE: Improvement in gene therapy carriers key step in hemophilia…

This video from Osmosis Health & Medicine describes hemophilia and what it really means to have the blood disorder. MORE: Daily infusion of low-dose factor VIII found to benefit some with hemophilia A.  With detailed illustrations, the video’s narrator explains that hemophilia comes from the words “love”…

First results from the Phase 3 HAVEN 2 study evaluating the effectiveness and safety of emicizumab in children younger than 12 with hemophilia A look promising. Roche announced that preventive treatment with emicizumab caused a reduction of the number of bleeds over time, with no major adverse events reported so…

Novo Nordisk A/S has launched HemaGo XChange, a new web-based service portal for people with bleeding disorders. This platform will address the overwhelming demands that patients with hemophilia might face in the monitoring and sharing of information about their disease. HemaGo XChange allows patients to monitor and share treatment…

Ixinity (recombinant factor IX product, IB1001) was found to be safe and effective in previously treated children with hemophilia B younger than 12, according to data from Aptevo Therapeutics‘ ongoing Phase 3 clinical trial. The company presented data from the analysis of two studies at the recent Hemostasis and Thrombosis…

San Francisco-based Catalyst Biosciences has completed toxicology studies of its new drug CB 2679d/ISU304 — a highly potent next-generation coagulation factor to treat hemophilia B. This  follows the therapy’s recent Investigational New Drug (IND) approval, a necessary step before starting clinical studies, by the Korean Ministry of Food and Drug Safety. Patients…