Alice Melão,  —

Shire is developing a potential new strategy to improve the effectiveness of genetic delivery of clotting factor VIII (FVIII) for the treatment of patients with hemophilia A, and clotting factor IX (FIX) for patients with hemophilia B. The most recent findings on these new therapeutic strategies and the latest…

An international team has joined efforts to establish guidelines for effectiveness and outcome measurements regarding gene therapies in hemophilia. The CoreHEM project will be led by researchers from McMaster University in Ontario, Canada, in collaboration with the National Hemophilia Foundation (NHF) in the U.S. and the Green Park…

Novo Nordisk’s recombinant coagulation factor IX nonacog beta pegol (N9-GP), under the U.S. trade name Rebinyn, has been approved by the U.S. Food and Drug Administration (FDA) to treat adults and children with hemophilia B. The company expects to launch this new treatment in the United States early in 2018. The…

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…

Dimension Therapeutics‘ gene therapy candidate drug DTX101 shows promising results by improving the levels of blood clotting factor IX (FIX) in adult patients with moderate/severe to severe hemophilia B, the company said. Hemophilia B is caused by FIX deficiency, due to mutations on the gene that encodes this…

Recent studies have addressed the importance of preventive, or prophylatic, treatment in severe cases of hemophilia, and its long-term benefits for children. The results may help establish guidelines and treatment recommendations leading to improved care for such kids. Both the World Health Organization and the World Federation of Hemophilia urge preventive treatment…

A Phase 3 extension trial has confirmed Alprolix’s long-term ability to prevent bleeding episodes in hemophilia B patients, and its safety. Those in the B-YOND extension trial did not develop an immune response against Alprolix. Adults and adolescents were treated for three years, and children under 12 for 18 months. “These results…

Adult hemophilia patients use hospital emergency departments mainly due to cardiovascular events, while pediatric patients most frequently complain of injuries, according to researchers. U.S. emergency department (ED) visits by hemophilia patients had a national estimated cost of $60 million in 2012, the year studied. These observations resulted from the retrospective and…

A new delivery mechanism of mRNA therapy was found to efficiently reduce hemophilia B symptoms by correcting the protein deficiency that characterizes the disease in a mouse study. This potential new method might be a viable alternative for many clotting disorders. The study, “Systemic delivery of Factor IX messenger RNA…

A recent survey has shown that the use of appropriate standardized outcome measures of hemophiliac patient treatments in routine clinical practice are not the norm. But implementing these measures could promote more effective personalized therapeutic approaches and reduce treatment-related costs. Hemophilia is a complex disease with a major impact on the…