The U.S. Food and Drug Administration (FDA) has granted both fast track and rare pediatric disease designations to Expression Therapeutics’ investigational stem cell therapy for hemophilia A. Each of these designations is intended to speed development of a candidate therapy. This stem cell therapy was designed as a potentially…
News
Many women carrying hemophilia-related mutations are not receiving the medical attention they need, according to a large-scale multinational study. Despite being treated in established healthcare systems, these women, often referred to as hemophilia carriers, frequently remain undiagnosed and excluded from official patient registries. Researchers found that a lack…
Bemiltenase alfa, an experimental factor X (FX) activator, helped with on-demand bleeding control for people with hemophilia A and B who had developed inhibitors against standard factor replacement therapies, according to results from early clinical trials. Inhibitors are antibodies that may reduce the effectiveness of hemophilia treatments, making it…
Roctavian (valoctocogene roxaparvovec-rvox) was associated with fewer bleeding episodes and less need for preventive treatment, or prophylaxis, in most adult men with severe hemophilia A for up to seven years after a single infusion, according to final results from a completed clinical trial. The Phase 1/2 clinical…
A computer-aided diagnostic tool using artificial intelligence (AI) can accurately detect joint bleeds in adults with hemophilia, a study showed. The researchers said the findings may lead to a way for patients to obtain ultrasound images at home, rather than traveling to a facility. “The CAD [computer-aided diagnosis] system…
Regular and sustained physical exercise — including aerobic activities, strength training, and balance exercises — may help ease chronic pain, improve physical performance, and enhance quality of life in people with hemophilia of all ages, according to a review study. The researchers found that exercise is generally safe when…
World Hemophilia Day, an annual effort to raise awareness of hemophilia and other bleeding disorders, is on April 17, and this year, the focus is on the importance of a prompt, accurate diagnosis as a key step toward achieving good health for people…
Preventive treatment with Hemlibra (emicizumab) is associated with better bleeding control and lower medication costs compared with immune tolerance induction, or ITI, among children with severe hemophilia A with inhibitors, a new study in China found. The researchers noted that use of the injection therapy Hemlibra also led…
A single dose of the gene therapy Roctavian (valoctocogene roxaparvovec-rvox) continues to prevent bleeds and the need for preventive treatment (prophylaxis) for up to five years in most treated men with severe hemophilia A, according to final Phase 3 trial results. Activity of factor VIII (FVIII), the clotting…
Measuring levels of YKL-40 — a protein associated with inflammation and tissue remodeling — may help detect hemophilic arthropathy, the joint damage caused by repeated bleeding, in people with severe hemophilia A, a study found. The researchers said the results “suggest that YKL-40 has potential as a biomarker, with its…
