Despite the COVID-19 pandemic, BioMarin Pharmaceutical‘s application to the U.S. Food and Drug Administration (FDA) seeking approval of valoctocogene roxaparvovec, an experimental gene therapy for adults with severe hemophilia A, remains on track. During a conference call, the company also said that if approved, the…

The risk of spontaneous bleeding episodes in patients with severe hemophilia A receiving preventive treatment changes over time and can be influenced by treatment history and other patient-related factors, a study has found. As such, the choice of an individual preventive — or prophylactic — regimen should take…

Dr. Reddy’s Laboratories announced the U.S. launch of a generic version of DDAVP (desmopressin acetate), used to control bleeding in people with mild hemophilia A. The generic is available under the name Desmopressin Acetate Injection USP, 4 micrograms/mL, in a carton of 10 single-dose ampules…

Our hemophilia community has been here before. A novel virus — previously, it was HIV — hit the world, and our community was uncertain about how it would affect us. Patients were told to turn to doctors for help and guidance, but the information each patient received varied…

Switching from on-demand to preventive treatment — or prophylaxis — significantly reduces bleeding in adults with hemophilia A, including the elderly, a real-life study shows. The study, “Long‐term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A,”…

Catalyst Biosciences has received a European Union patent for its investigational hemophilia B treatments, including its lead therapy candidate, dalcinonacog alfa (DalcA) and a preclinical gene therapy candidate called CB…

Greater awareness of hemophilia and its symptoms is associated with better adherence to treatment by patients, and those with more severe disease are most likely to use a treatment as prescribed, a study based on a new questionnaire shows. Responses were seen to validate this questionnaire, which addresses…

Blood levels of two proteins — C-reactive protein (CRP) and vascular endothelial growth factor (VEGF) — involved in inflammation and blood vessel formation are significantly associated with, and may help predict, acute bleeding in the joints of people with severe hemophilia A, a study has found. The study,…

Data from a completed Phase 2b trial indicate that dalcinonacog alfa (DalcA) is an effective and safe therapy to prevent bleeding in people with severe hemophilia B. Final results from this open-label study in six patients are expected by June. “We are pleased to have successfully completed the DalcA Phase…

In the past month, it has been hard for me to focus on hemophilia advocacy efforts, which normally are a burning passion. Disappointment over an opportunity to work more deeply with the hemophilia community that didn’t pan out and COVID-19 realities have me pulling back.