The rate of intracranial hemorrhage (ICH) — a bleed in the brain — is higher in children and adults with hemophilia than in the general population, an Italian study suggests. ICH rates were particularly high among adults with mild disease and with high blood pressure. Most patients received only…

More than 80% of the men with severe hemophilia A treated with the investigational gene therapy Roctavian (valoctocogene roxaparvovec) in the Phase 3 GENEr8-1 trial remained bleed-free two years later, and nearly all were off standard preventive therapies. These findings were among new data presented at the recent 15th Annual…

Adults with severe hemophilia B have a persistent number of bleeds per year, as well as joint problems and mild to moderate chronic pain, revealing an unmet medical need in these patients, according to real-world data from the European CHESS II study. The study, “Clinical, humanistic, and economic…

In a Phase 1/2 dose-finding study called B-AMAZE, FLT180a gene therapy from Freeline Therapeutics brought about sustained levels of factor IX, the clotting factor that is missing in people with hemophilia B, and this translated into fewer bleeds each year. Levels of factor IX within the normal range…

Physical activities with a high risk of a collision are linked with a greater risk of bleeding in people with moderately severe hemophilia A, a six-month U.S. study reports. This risk was higher for people using on-demand versus preventive replacement factor VIII (FVIII) therapies, the researchers found, and they suggested…

Preventive treatment with Hemlibra (emicizumab) is safe and can effectively prevent bleeds in people with mild or moderate hemophilia A, according to an interim analysis of data from the HAVEN 6 clinical trial. Results will be presented at the annual meeting of the American Society of Hematology (ASH)…

When given monthly to people with hemophilia A or B with inhibitors, fitusiran significantly reduced bleeding, and for some, fully eliminated them, according to data from the Phase 3 ATLAS-INH study.

Hemophilia patients experienced improvements in joint health, mobility, and overall quality of life after completing a supervised eight-week exercise program, a study reports. However, in order to achieve long-lasting benefits, regular exercise may be required. The study, “Effects of a supervised therapeutic exercise program on musculoskeletal health and…

The World Federation of Hemophilia (WFH) is commemorating having successfully distributed, since 1996, over one billion international units (IUs) of factor and non-factor replacement therapies for people with inherited bleeding disorders, such as hemophilia, through its Humanitarian Aid Program. Donations from the program have improved the quality…

Differences in sensory detection and pain thresholds were found in hemophilia patients taking prophylactics, compared to healthy individuals, and these differences did not change over the course of a one-year follow-up study. These findings indicate that “under prophylactic treatment, the existing difference in the pain profile between [people with…