Eptacog beta was safe and almost 100% effective at stopping bleeds within 24 hours in boys ages 12 and younger with hemophilia A or B, according to final data from the Phase 3 PERSEPT 2 trial. According to researchers, this bypass agent may be an important option for…

Factor VIII (FVIII) levels in the bloodstream of men with severe hemophilia A treated with the investigational gene therapy Roctavian appear to be influenced by regulatory molecules involved in F8 gene activity and FVIII protein folding and release, a study reveals. Researchers also found a dose-dependent relationship between…

Bone mineral density — the amount of minerals in bone tissue — is markedly lower in children with moderate and severe hemophilia than their healthy peers as a result of recurrent bleeding episodes that damage their joints, according to a small, single-center study in India. Preventive, or prophylactic, treatment was…

Boys with severe hemophilia A who switched to Kovaltry (octocog alfa), which replaces a missing clotting protein called factor VIII (FVIII), retained the therapy for longer in the body and had fewer bleeds, a study in China reported. Findings come from a head-to-head comparison of Kovaltry and three…

Hemophilia B patients who switched to Rebinyn (nonacog beta pegol), an extended half-life factor IX (FIX) replacement therapy, had similar or better responses than with their previous standard or extended half-life FIX replacement therapies, a real-world study in Canada suggested. The study, “Switching to nonacog beta…

Excessive bleeding after oral surgery led to two people in China, both with normal pre-operative blood clotting tests, being diagnosed with hemophilia A, according to a recent case report. Mild hemophilia can go undetected in standard laboratory tests, and oral surgeons need to be prepared for such cases, the research…

Elevated levels of the immune signaling protein BAFF were found in the bloodstream of boys who developed neutralizing antibodies against FVIII replacement therapy, a standard treatment in hemophilia A, a study reported for the first time. Changes in the gene that encodes the BAFF protein, involved in the growth…

Weekly use of the experimental, long-acting therapy efanesoctocog alfa safely and effectively prevents bleeds in adolescents and adults with severe hemophilia A, according to top-line, one-year data from the Phase 3 XTEND-1 trial. The therapy — being jointly developed by Sanofi and Sobi — was also superior to approved preventive therapies…

BioMarin Pharmaceutical has completed enrollment in GENEr8-3, a Phase 3b study evaluating its investigational gene therapy Roctavian (valoctocogene roxaparvovec) in combination with corticosteroids in men with severe hemophilia A. Top-line data from the 52-week (one year) study are expected to be released in early 2023, according to a…

In Austria, most patients with hemophilia have been vaccinated against COVID-19 — but some expressed concerns over the vaccine’s potential side effects — a small study found. Data showed that 75.0% of the patients in the study were fully vaccinated and that 87.5% had received at least one dose…