News

Jivi was safe and effective at preventing and treating bleeds for more than five years in boys under age 12 with severe hemophilia A, according to long-term data from a Phase 3 trial’s extension study. Data analysis also showed that most of…

For people with hemophilia B, switching to Rixubis (nonacog gamma) as a factor IX (FIX) replacement therapy was not associated with increased bleeding rates, dose changes, adverse blood clotting events, or the development of inhibitors among patients with moderate to severe forms of the disease,…

A research study is asking people with hemophilia and other inherited bleeding disorders living in the U.K., U.S., or Germany to take part in a survey evaluating how the COVID-19 pandemic is affecting the mental health of those with these diseases. The four-part survey, which can be…

Mild-to-moderate hemophilia has a significant impact on the physical and psychosocial health, and life quality, of both men and women, according to a recent study. Findings also highlighted the unmet needs of these patient groups, supporting better guidelines and quality of care for people with mild-to-moderate disease, its researchers…

Enzyre is partnering with Takeda with a goal of developing at-home assays to help people with monitoring congenital bleeding disorders, including hemophilia, and to diagnose these disorders. This partnership builds on an existing, 2019 research collaboration between these…

The National Hemophilia Foundation (NHF) is calling for nominations for its Awards of Excellence, which honor members of the bleeding disorders community. The awards are for those who have helped improve the lives of patients with bleeding diseases, including those with hemophilia, through treatment and care, advocacy,…

The U.S. Food and Drug Administration (FDA) has given regenerative medicine advanced therapy (RMAT) designation to Roctavian, an investigational gene therapy by BioMarin Pharmaceutical for severe hemophilia A. The designation is “a critical program to advance the efficient development and regulatory review of regenerative medicine products that have the…

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…

The European Medicines Agency (EMA) has agreed to review an application by Laboratoire Francais du Fractionnement et des Biotechnologies, a French biopharmaceutical known as LFB, requesting the approval of eptacog beta for the treatment of bleeding episodes in patients with hemophilia A or B with inhibitors. A…

Activities are underway to mark Bleeding Disorders Awareness Month, set aside each March to call attention to such disorders as hemophilia and von Willebrand disease, and the more than three million U.S. residents living with them. Patients, caregivers, and activists nationwide are jumping on social media platforms, taking…