News

AMT-061 (etranacogene dezaparvovec), uniQure’s investigational gene therapy for hemophilia B, likely did not cause the case of hepatocellular carcinoma (HCC) — a form of liver cancer — that led to the program’s clinical hold in December 2020, an investigation has found. The U.S. Food and Drug Administration (FDA) had put the AMT-061…

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

Children and teenagers with hemophilia are considerably more physically active than adult patients, possibly reflecting more recent emphasis on the benefits of exercise and the arrival of better treatments, according to a recent review study. Yet, how physically activity relates to bleeding events remains unclear, its researchers wrote, and long-term…

Both swimming and hydrotherapy can improve functional capacity in hemophilia patients, according to data from a recent study. Of those two types of aquatic exercises, however, hydrotherapy was associated with the most significant functional improvements in these patients. Those findings were reported in the study, “Aquatic…

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,…