News

An immersive multimedia exhibit, which features portraits by renowned photographer Rankin, seeks to educate about life with hemophilia and the disease community’s hope for the future. The exhibit, called “Portraits of Progress,” is a collaboration between biotherapeutics company CSL Behring and Rankin, a British photographer who has done…

Dosing has begun in a second group of hemophilia B patients enrolled in a Phase 1/2 dose-confirmation trial of the experimental gene therapy FLT180a, the treatment’s developer, Freeline, announced. The dose determined to be optimal in this study is expected to finalize plans for a pivotal Phase 3…

The levels of blood clotting factors, physical activity, and bleeding history are all potential risk factors for future bleeds in hemophilia patients under preventive therapy, a small review study from Canada suggests. “These and other risk factors for bleeding could be combined in a risk assessment model (RAM) for…

One year of treatment with Elocta (efmoroctocog alfa) — an extended half-life replacement therapy known as Eloctate in the U.S. — reduced the frequency of bleeds in the elbows and knees of adults with severe hemophilia A, a Spanish study showed. Reduced pain intensity in the elbows also…

Children and young adults with hemophilia A and hemophilia B have several behavioral problems, ranging from depression and anxiety to aggressive behaviors, a small study suggests. Age, disease severity, and joint disease duration were found to be significantly correlated with several of these problems. The findings were reported in the…

Preventive treatment with a low dose of factor replacement therapy in children with hemophilia A led to sufficient levels of the clotting factor in most patients, but the time in which it did so varied, a recent study reported. Researchers noted that while findings support the use of this…

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to efanesoctocog alfa, an experimental replacement therapy for hemophilia A designed for once-weekly dosing. This designation is given by the FDA to help speed the development and review of treatments for serious or life-threatening conditions. Specifically,…

A single dose of the experimental gene therapy Roctavian (valoctocogene roxaparvovec) prevents bleeds and the need for preventive treatment, or prophylaxis, for up to six years in men with severe hemophilia A, according to updated data from a Phase 1/2 trial. “With every year of observation in this…

The gene therapy BBM-H901 increased factor IX activity in 10 men with moderate to severe hemophilia B, while reducing bleeds and the use of FIX replacement therapy over one year, according to results of a pilot Phase 1 trial. “The study was the first clinical trial conducted by Belief BioMed…

The U.S. Food and Drug Administration (FDA) has accepted — under priority review — a marketing application for EtranaDez (etranacogene dezaparvovec), an investigational gene therapy for adults with hemophilia B. The FDA grants priority review to investigational therapies designed to treat serious medical conditions. If approved, the treatment…