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 release. The late-breaking…

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…