The U.S. Food and Drug Administration (FDA) has approved Expression Therapeutics‘ request to open a Phase 1 trial of ET3, an investigational gene therapy for people with hemophilia A. The decision followed a review of the company’s investigational new drug application (IND) requesting clearance of ET3…

A single dose of the experimental gene therapy AMT-180 continues to be well-tolerated and to enhance blood clotting activity independent of factor VIII levels in mouse and primate models of hemophilia A, a study shows. Titled, “Human Dose Prediction of a Novel Factor IX Variant Gene Therapy…

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…

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,”…

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,…

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.

Treatment with Esperoct (turoctocog alfa pegol) effectively prevents bleeding during and after major surgery in patients with severe hemophilia A, final data from a Phase 3 trial show. The study, “…

In contrast to observations in the general population, blood type and levels of von Willebrand factor (VWF) don’t appear to influence factor VIII (FVIII) levels in people with non-severe hemophilia A, a study reports. Moreover, age was found to play only a minor role in FVIII levels in…