News

Factor VIII (FVIII) levels in the bloodstream of men with severe hemophilia A treated with the investigational gene therapy Roctavian appear to be influenced by regulatory molecules involved in F8 gene activity and FVIII protein folding and release, a study reveals. Researchers also found a dose-dependent relationship between…

Bone mineral density — the amount of minerals in bone tissue — is markedly lower in children with moderate and severe hemophilia than their healthy peers as a result of recurrent bleeding episodes that damage their joints, according to a small, single-center study in India. Preventive, or prophylactic, treatment was…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

Osteoporosis and other bone disorders are more common in hemophilia carriers than in the general population, according to an analysis of U.S. insurance data. Its researchers also found a higher rate of bone problems among people with von Willebrand disease (VWD), another inherited bleeding disorder. “This study highlights the need…

Healthcare costs of a hospitalization for children and adults with hemophilia in the U.S. are higher than for those who do not have a bleeding disorder, a study reported. Hospitalizations for children with hemophilia were also seen to be mostly driven by bleeding and catheter-related infections, while high blood pressure and other…

Boys with severe hemophilia A who switched to Kovaltry (octocog alfa), which replaces a missing clotting protein called factor VIII (FVIII), retained the therapy for longer in the body and had fewer bleeds, a study in China reported. Findings come from a head-to-head comparison of Kovaltry and three…

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…

Hemophilia B patients who switched to Rebinyn (nonacog beta pegol), an extended half-life factor IX (FIX) replacement therapy, had similar or better responses than with their previous standard or extended half-life FIX replacement therapies, a real-world study in Canada suggested. The study, “Switching to nonacog beta…

Excessive bleeding after oral surgery led to two people in China, both with normal pre-operative blood clotting tests, being diagnosed with hemophilia A, according to a recent case report. Mild hemophilia can go undetected in standard laboratory tests, and oral surgeons need to be prepared for such cases, the research…

Serious bleeds are often a fatal complication in people with hemophilia if not managed early, a 10-year study from Senegal shows. These findings highlight the need for prompt diagnosis of severe bleeding and early administration of replacement therapy to lower the risk of mortality. In resource-limited countries, increasing patients’ and…