Two new mutations affecting the production of functional clotting factor VIII were identified in a group of Vietnamese patients with hemophilia A, researchers report. Their findings were described in a letter to the editor, “Mutation characteristic of 103 haemophilia A patients in Vietnam: Identification of novel mutations,”…

Women who are hemophilia carriers are at a higher risk of experiencing heavy bleeding after giving birth, particularly those who are not on a prophylactic, or preventive, treatment regimen, a new review study has found. Researchers say these findings indicate that more intensive preventive treatments might benefit carriers about…

People living with hemophilia face a 46% higher risk of bone fractures than the general population, with that risk increasing for those with severe disease or a history of treatment-blocking antibodies. These are the findings of a large-scale Canadian study that highlights a growing health concern for patients as…

I hate it when the term “hero” is used to describe me and my daily life with hemophilia. Honestly, I despise it being used to describe anyone living with a rare disease or disability. I think it’s the wrong word, as its use can inadvertently minimize the emotional challenges…

Medexus Pharmaceuticals has completed patient enrollment in its clinical trial investigating Ixinity as a preventive treatment for children younger than 12 with hemophilia B.  The open-label Phase 4 trial (NCT03855280) achieved its target enrollment goal of 22 participants and is being conducted in 15 centers in Brazil,…

For many millennial families, the dining table has become somewhat of a relic. What was once a central feature of family life — where meals were shared, stories told, and connections made — is now increasingly overlooked. It’s a trend I’ve seen in many homes, including my own,…

Treatment with the experimental gene therapy fidanacogene elaparvovec led to significant reductions in bleeding rates for men with moderately severe to severe hemophilia B who participated in a Phase 3 clinical trial. That’s according to top-line data from the study BENEGENE-2 (NCT03861273), recently announced by the therapy’s developer,…

A person in Germany with severe hemophilia A has been treated with Roctavian (valoctocogene roxaparvovec-rvox), the first time the gene therapy has been used commercially in Europe, according to BioMarin Pharmaceutical, its developer. “Today represents an important milestone for the hemophilia community and for patients and…

The 1980s were my favorite decade. I was in high school, and my music was the most important thing in my life. I practiced hard for auditions in the hopes of making the Texas All-State Band. Life was good. My perception of that decade changed after my first…