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…