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…

To help prevent bleeds in hemophilia patients, many people living with the disease need to have infusions of clotting factor. In this video from infuzrTV, we meet super cute 5-year-old Brandston. Brandston has severe hemophilia A and needs to have clotting factor infusions on alternate days. MORE: …

In a small study, children with hemophilia A had distinct changes in certain immune cells and inflammatory signaling molecules compared with healthy children, and the type of preventive treatment they receive may shape those immune responses in different ways, according to the researchers. In lab experiments, macrophages — immune…

Over the past few years, I have had the pleasure of connecting with many women who are diagnosed with hemophilia A, hemophilia B, or as symptomatic carriers. These women have so many stories to share. The thread of commonality in their journeys is their struggle…

Cell therapy with endothelial progenitor cells and stem cells genetically engineered to produce a functional clotting factor VIII (FVIII) may provide a stable and long-term treatment for hemophilia A, a mouse study has found. The study shows that the transplant of cells…

Precision BioLogic has developed what it says is a better way of detecting levels of substances in hemophilia A patients’ blood that prevent clotting factor therapies from working as well as they should. It collaborated with Roche and Genentech in creating a kit it says can detect levels of the…

An experimental prenatal cell and gene therapy designed to restore production of the missing clotting protein in hemophilia A showed maternal safety signs in a U.S. study in sheep. The therapy, called PLC-mcoET3, is designed to transplant genetically engineered cells into the fetus so they can produce factor VIII…

Long-term treatment with Novoeight (turoctocog alfa) is safe and effective at preventing bleeding episodes in patients with hemophilia A of all ages who had already received prior treatment, a Phase 3b extension trial shows. The study, “Long‐term safety and efficacy of turoctocog alfa in prophylaxis and treatment…

Swedish Orphan Biovitrum (Sobi) announced the successful enrollment of the first hemophilia A patient in the company’s ReITIrate clinical trial. The Phase 4 study (NCT03103542), sponsored in collaboration with Bioverativ Therapeutics, is actively recruiting participants who have developed inhibitors and who have failed to respond to other therapies. Researchers will…

“There are only two ways to live your life. One is as though nothing is a miracle. The other is as though everything is a miracle.” — often attributed to Albert Einstein I live with a bleeding disorder, hemophilia A. Survival often is truly about attitude, optimism, and perseverance.