News

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD), held…

A Phase 1/2 trial (NCT03520712) testing BioMarin Pharmaceutical’s investigational gene therapy valoctocogene roxaparvovec in severe hemophilia A patients with pre-existing AAV5 antibodies has dosed its first participant. “Administration of valoctocogene roxaparvovec to this first patient seropositive for the AAV5 capsid is an important next step in our plan to…

The National Heart, Lung and Blood Institute (NHLBI) is hosting a State of the Science Workshop on May 15-16, 2018, in Bethesda, Maryland, to include input from the hemophilia community into a coordinated strategy for future basic, translational, and clinical research. Although preregistration is closed, on-site registration…

Teaching hemophilia patients who have undergone joint replacement surgery how to administer their intravenous (IV) self-infusions in sterility, can decrease the rate of infections post-surgery significantly, a six-year education program shows. The program outcomes will be shared at the upcoming World Federation of Hemophilia 2018 World Congress May 20-24…

In recognition of World Hemophilia Day 2018, the biopharmaceutical company Bioverativ is sharing the stories of people with hemophilia in the developing world to highlight the life-changing potential of adequate treatment. In honor of this year’s theme, “Sharing Knowledge Makes Us Stronger,” Bioverativ will collaborate with members of the hemophilia community…

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.