A team of U.S. scientists discovered a naturally occurring F8 gene mutation that leads to increased activity of the factor VIII (FVIII) coagulation factor and enhanced blood clotting, offering new insights into the treatment of hemophilia A. The mutation was identified in a young man susceptible to excessive blood…

In a real-world study in South Korea, preventive treatment with Adynovate (rurioctocog alfa pegol) was shown to be effective at preventing bleeding episodes among most children and adults with hemophilia A  — findings that line up with those from clinical testing. In fact, “76.1% of patients on prophylactic…

“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.

Scientists have developed advanced liver organoids — lab-grown “mini-organs” that mimic the function of organs in the body — that are capable of growing their own functional blood vessels and producing clotting factors that could eventually be used to treat hemophilia, according to a study. In a mouse model…

In people with hemophilia A, certain immune markers in the blood may help predict patient responses to immune tolerance induction (ITI) — a treatment regimen used for individuals who develop inhibitors after factor replacement therapy — according to a new study by researchers in Brazil. Inhibitors, neutralizing antibodies…

People with hemophilia A in China see major health challenges, such as frequent joint bleeding, joint malformation, and high rates of hospital visits, primarily owing to the limited use of preventive therapies, a real-world study shows. After a bleeding episode, most patients rely on on-demand treatment, which is less…

A lack of family history of disease can delay a diagnosis and timely initiation of preventive therapy in children with hemophilia A, increasing the need for intensive factor replacement therapy when a first bleeding event happens, a study shows. “These observations highlight the importance of an early diagnosis in…

CAR T-cell therapy, a type of treatment that weaponizes the body’s immune system and was originally developed to treat cancer, for the first time successfully treated a patient with acquired hemophilia A (AHA), a study reported. The study, “Anti-CD19 CAR-T cell therapy for acquired hemophilia A,”…

The blood-clotting protein factor VIII (FVIII) that’s missing or faulty in people with hemophilia A is flexible and adopts multiple shapes, some of which favor the binding of neutralizing antibodies that can reduce the effectiveness of FVIII replacement therapies, researchers have learned. “This helps explain why some people…