Patricia Inácio, PhD, science writer —

GeneVentiv Therapeutics has received a $2.5 million grant to conduct further preclinical tests on its one-time infusion gene therapy for hemophilia A and B patients with or without clotting factor inhibitors. The therapy, GENV-HEM, will be tested in a large study with a canine model of hemophilia, following…

Men with severe hemophilia who wore activity wristbands to self-manage their physical activity saw significant improvements in their physical health and were capable of spending more time on moderate-intensity physical activity, a small study from Spain has found. According to the researchers, these findings demonstrate that activity wristbands “can…

Progressive joint disease caused by repeat bleeds, a condition known as hemophilic arthropathy, affects people with moderate hemophilia in Nordic countries regardless of age, commonly causing them pain and restricting their mobility, a study found. Middle-age patients, those 41 to 50 years old, felt their overall quality of life…

The development of neutralizing antibodies, or inhibitors, against the clotting factor in replacement therapies administered to people with hemophilia may depend on the type of product used, a recent study has found. In previously untreated people with severe hemophilia A, or those who lack…

When given as a prophylactic, or preventive therapy, or on an on-demand basis, FEIBA (factor eight inhibitor bypassing activity) was deemed safe and effective at lessening the number of bleeds in people with severe hemophilia and high levels of inhibitors. That’s according to four-year data from a real-world…

A 48-year-old man with severe hemophilia A developed a rare intramural hematoma with blood leaking into one of the layers of the intestine wall that was successfully treated with an early infusion of the missing blood clotting factor, a recent case report described. Prophylactic treatment prevented its recurrence. A…

Most people with hemophilia responding to a survey in the Netherlands were generally satisfied with their treatment care. Patients’ interactions with healthcare professionals, availability of care, and coordination were cited as the most important factors for satisfaction. However, they reported improvements were needed regarding information about new treatments, their…

People with hemophilia showed no heightened protection against COVID-19 severity or mortality, a large retrospective U.S. outcomes study found. “Having hemophilia was not a risk factor for death,” the researchers wrote, noting, however, that hemophilia patients “were more likely to have moderate COVID-19 severity and to be hospitalized.” Older…

2seventy bio announced that it has achieved a key preclinical milestone under its joint program with Novo Nordisk to develop a gene-editing tool to treat hemophilia A. Details of the “proof of concept” research that met this milestone — reported to be worth $15 million to the…

Patient dosing has been completed in a registrational Phase 3 clinical trial in China testing BBM-H901, Belief BioMed‘s (BBM)’s investigational gene therapy for hemophilia B, the company announced. BBM-H901 is designed to restore the production of factor IX (FIX) — the blood-clotting protein that is faulty or missing…