Metagenomi is now working on preclinical studies to advance MGX-001, its experimental gene-editing therapy for hemophilia A, with the goal of bringing the treatment candidate into clinical trials in 2026. As part of a corporate update also noting 2024 progress, Metagenomi announced that, this year, it will be…

People with severe hemophilia A who switch from standard to extended half-life replacement therapies experience fewer bleeds and require less frequent infusions, an analysis of patients in Austria indicates. Whether these gains are reflected in lower overall treatment costs, however, is less clear, its researchers added. Data from this…

Becoming a parent changes everything in life. This was certainly true for my husband, Jared, who’d always dreamed of being a father, despite having severe hemophilia B and frequent seizures. When our daughter, Cittie, was born, that dream came to life, giving him more purpose and even improving…

Researchers have found  that N8-GP (turoctocog alfa pegol) treatment improved the quality of life and satisfaction of children with severe hemophilia A. The study titled “Health-Related Quality of Life and Satisfaction of Children with Severe Hemophilia a Treated with Turoctocog Alfa Pegol (N8-GP),” appeared in the journal Blood.

An interview-based study from Japan found professional support was necessary when mothers inform their daughters they might be carriers for hemophilia, as the women found themselves “not knowing what to do” and expressing “anxiety due to uncertainty.” In Japan, parents are often responsible for telling a child that a genetic…

Less-frequent and easier administration of factor VIII (FVIII) — the clotting protein that is missing or defective in people with hemophilia A — is likely to boost treatment adherence among patients with severe disease, a South Korean study has found. The study, “Patients’ and parents’…

Casebia’s two-stage gene therapy approach was successful in delivering the human gene coding for clotting factor VIII (FVIII) in a mouse model of hemophilia A, leading to stable and increasing levels of the factor, researchers said. “Our scientific team, led by Alan Brooks, Ph.D., have achieved a notable…

The World Federation of Hemophilia states that any person, male or female, with factor levels below 40 percent has hemophilia. However, conventional medical wisdom has not caught up. Women who have abnormal bleeding should be assessed for hemophilia, particularly when they have a family history. Yet too…