Investigational gene therapy SB-525 is well-tolerated and leads to a dose-dependent increase in factor VIII (FVIII), according to interim results from eight patients with severe hemophilia A enrolled in the Phase 1/2 Alta clinical trial. Data showed that patients who received the highest dose of SB-525 — 3e13 vg/kg dose — reached…

Hemophilia is it a genetic disorder where the body doesn’t produce clotting factor, which means blood can’t clot. This makes patients susceptible to both heavy external and internal bleeding, which if left untreated, can be life-threatening. Thanks to Hemophilia Village, we have the answers to some of the most commonly…

Efanesoctocog alfa, a long-lasting factor VIII (FVIII) replacement therapy, has now been approved in the European Union for hemophilia A. It will be marketed under the brand name Altuvoct. Altuvoct can be used as an on-demand treatment for bleeding episodes, a once-weekly prophylactic (preventive) therapy to reduce the…

Voncento — a highly concentrated mixture of Von Willebrand factor (VWF) and clotting factor VIII (FVIII) — was seen to safely and effectively treat or prevent bleeds in children, age 12 and younger, with severe hemophilia A, according to data from a Phase 3 trial. Findings were reported in the…

In real-world settings, Hemlibra (emicizumab-KXWH) can safely control bleeds in children with hemophilia A better than previous treatment regimens, a study reports. Hemlibra’s improved efficacy was more pronounced among patients with inhibitors, or neutralizing antibodies targeting factor VIII (FVIII), which is the clotting protein missing in people with…

The U.S. Food and Drug Administration (FDA) has cleared for laboratory use CRYOcheck Factor VIII Deficient Plasma with VWF, a way of detecting factor VIII (FVIII) deficiency in blood samples. Now available in the U.S., Precision Biologic’s manufactured plasma — the liquid part of blood that…

Gene constructs making up Roctavian, an investigational gene therapy for hemophilia A, did not permanently integrate into the genome of non-human primates or cause any signs of tumors or malignancy in the animals.  These are…

Mr. Man started coughing on Friday and grew much worse by Saturday. My boy struggled with flu-like symptoms all weekend. We thought that by giving him allergy meds and cough suppressants, he would recover and be well for school on Monday. No such luck. The medicines wouldn’t work,…

The U.K.’s National Institute for Health and Care Excellence (NICE) has recommended Altuvoct (efanesoctocog alfa) be available through the National Health Service (NHS), England’s public healthcare system, as a treatment option to manage and prevent bleeds in people with severe hemophilia A, ages 2 and older. This decision…

The levels of sclerostin, a protein that regulates bone formation, may be linked to disease severity and reflect bone alterations in children with hemophilia A, a study suggests. The study, “Serum Sclerostin Level and Bone Mineral Density in Pediatric Hemophilic Arthropathy,” was published in The Indian…