News

A newly identified class of stem cells in blood vessels, called vascular endothelial stem cells, were able to repair injured vessels and ease bleeding symptoms in a mouse model of hemophilia, researchers at Osaka University in Japan report, suggesting these cells may treat this bleeding disorder. The vascular endothelial stem cells, or VESCs,…

The cost of hemophilia treatment doubled for patients who switched from standard half-life (SHL) to extended half-life (EHL) products, according to a recent study by Prime Therapeutics. The findings of the study were presented at the Academy of Managed Care Pharmacy’s (AMCP) Managed Care & Specialty…

Gene therapy delivered to muscle was able to completely correct hemophilia B deficient blood clotting in a safe and well-tolerated manner for several years — in animal models of the condition. The study with finding, “Complete correction of hemophilia B phenotype by FIX-Padua…

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

Magellan Rx Management, the pharmacy benefit management division of Magellan Health, recently shared results from the first six months of its hemophilia management program as it observed World Hemophilia Day April 17. The focus of the hemophilia management program is to reduce unnecessary costs while improving quality of care.

CSL Behring marked World Hemophilia Day April 17 by shipping more than 10 million international units (IUs) of its medicines to treat hemophilia A to developing countries through the nonprofit World Federation of Hemophilia (WFH). The medicines were sent to countries like Vietnam, Cameroon and others through the WFH’s…

The U.S. Food and Drug Administration granted breakthrough therapy designation to Genentech’s Hemlibra (emicizumab-kxwh) for people with hemophilia A without factor VIII inhibitors. Hemlibra is designed to bring together the proteins required to activate the natural coagulation cascade and restore the blood clotting process for patients with…

The Korean Ministry of Food and Drug Safety has agreed to Catalyst Biosciences’ request to add a sixth group to a Phase 1/2 clinical trial of its hemophilia B treatment. The sixth group in the trial (NCT03186677) of the therapy that Catalyst calls CB 2679d (ISU304) will include up…

Hemophilia A patients who do not refrigerate their bleeding prevention therapy are more satisfied with it and report fewer restrictions on their daily activities, a study finds. Researchers also discovered that most of the patients were unaware that they could keep their recombinant factor VIII clotting therapy at room temperature.

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. The Office of Rare Diseases Research (ORDR), headquartered in Bethesda, Maryland, was established in 1993 within the NIH Office of the…