The U.S. Food and Drug Administration (FDA) has approved Shire’s Adynovate for the treatment of pediatric patients under 12 years of age with hemophilia A. Adynovate also was approved for use by both adults and children undergoing surgical procedures, to control bleeding episodes. Adynovate is a recombinant Factor…

Biogen recently hired two more executive management team members for Bioverativ, a Biogen spin-off company that is expected to be up and running in early 2017. Bioverativ will focus on the discovery, research, development, and commercialization of new treatments for hemophilia and other blood disorders. Rogerio Vivaldi, MD, is Bioverativ’s new executive…

NuFACTOR is accepting applications for the 2017 Annual Eric Dostie Memorial College Scholarship. NuFACTOR is a specialty pharmacy of FFF Enterprises, a distributor of plasma products, vaccines and biosimilars, among others. NuFACTOR specializes in the treatment of chronic conditions in several areas of medicine, including blood disorders and…

Genentech will present more than 60 abstracts at the 58th American Society of Hematology Annual Meeting (ASH 2016). Nine of Genentech’s approved or investigational medicines will be featured, including Gazyva (obinutuzumab), Rituxan (rituximab), Venclexta (venetoclax) and emicizumab, the company’s investigational drug in hemophilia. More specifically, Genentech will present…

Scientists have improved protein drugs using an approach based on “ancestral sequence reconstruction,” or ASR. The newly established strategy promises to enhance the ability to engineer more efficient and durable proteins, as in the case of human factor VIII for hemophilia. The study “Enhancing the pharmaceutical properties of protein drugs by…

Researchers reviewed the use of high-purity concentrates containing two plasma molecules that are lacking in patients with Von Willebrand disease (VWD), a hemorrhagic disorder. These products may be a good strategy for managing pediatric patients with VWD and hemophilia A (HA) who do not respond to other treatments. The study, “Human Von…

Researchers analyzed the patterns of bruising in children younger than six years old to understand the extent to which their inherited bleeding disorders, such as hemophilia, affected the number, size, and location of these bruises — and found that bruising in the youngest of these children may be an early…

San Rafael, California-based BioMarin Pharmaceutical will present interim data of an open-label, Phase 1/2 clinical trial of BMN 270, an investigational gene therapy treatment for hemophilia at the upcoming XXXII International Congress of the World Federation of Hemophilia (WFH) July 24-28 in Orlando, according to a news…

Researchers investigated self-reported health-related quality of life (HrQoL) in children and adolescents with hereditary bleeding disorders, such as hemophilia A, and found no differences between patients and their healthy siblings and peers. The research paper, titled “Health-Related Quality of Life in Children and Adolescents with Hereditary Bleeding Disorders…

Researchers at the Birjand University of Medical Sciences and colleagues investigated the immunity status of hemophilic patients against hepatitis A, and the need for hepatitis A vaccination in that patient population. Findings indicate that a significant proportion of hemophilic patients under age 20, who were evaluated in the study, had no immunity…