Mobility problems, bleeding, pain, and uncertainly in daily life are the biggest challenges people with hemophilia are facing, according to a survey from six central European countries. Additionally, while most respondents, including caregivers, felt well informed on hemophilia, they continue to seek further education on the condition and new…

Gene constructs making up Roctavian, an investigational gene therapy for hemophilia A, did not permanently integrate into the genome of non-human primates or cause any signs of tumors or malignancy in the animals.  These are…

Severe hemophilia B patients receiving prophylactic, or preventive treatment with factor IX face a significant financial and personal burden, according to data from two U.S. population-based medical databases. These findings were reported in a study, “Clinical, humanistic, and economic burden of severe hemophilia B in the United States:…