José Lopes, PhD, managing science editor —

Combining two gene editing approaches in cells derived from a patient with severe hemophilia A successfully restored the levels and activity of the faulty clotting factor VIII (FVIII) and reduced bleedings in a mouse model of the disease, according to a new study. The research, “ssODN-Mediated…

Less frequent infusions with Jivi led to fewer bleedings, a greater ability to engage in physical and workplace activities, less vein damage, and better emotional well-being in men with severe hemophilia A compared to previous therapies used, participants in a Phase 2/3 trial largely agreed. “Exploring the Impact of…

Inadequate access to treatment centers, lack of expert care, and high bleeding rates lead to similar joint complications in hemophilia patients with and without inhibitors, and joint problems in 70% of the people taking part in a recent study in five developing countries. The research, “HAEMOcare:…

The ratio between the blood clotting protein factor VIII (FVIII) and the von Willebrand factor (VWF) may be a reliable biomarker of recovery and relapse in patients with acquired hemophilia A (AHA), a study suggests. The research, “The factor VIII:C/VWF:Ag ratio as a useful tool to…

Fewer HIV infections and better care may explain the higher prevalence of hemophilia in male patients in Indiana between 2011 and 2013 than previously reported in six other states, according to a new study. The findings also revealed a higher utilization of Hemophilia Treatment Centers (HTCs) by this patient…

Treatment of hemophilia A with bypassing agents is associated with significantly higher costs than with factor replacement therapy, according to a real-world study in the U.S. The study, “Healthcare costs among patients with hemophilia A treated with factor replacement or bypassing agents,” appeared in…

Regular preventive treatment is able to at least partially prevent bleeding episodes in adolescent and adult males with severe hemophilia A and without inhibitors, according to a real-world study. The study, “Bleeding and safety outcomes in persons with haemophilia A without inhibitors: Results from a prospective…

A Phase 3 clinical trial of treatment candidate AMT-061 for severe and moderately severe hemophilia B has treated its first patient. The open-label HOPE-B study (NCT03569891) is testing the safety and effectiveness of AMT-061 in adult men with hemophilia B. Patient recruitment is continuing for an estimated total of 56…

Repetitive movements, fitness level, and prolonged and competitive physical activity are among the factors that raise bleeding risks for people with hemophilia taking part in such activities, a study based on a survey of physical therapists in the U.S. reports. Duration of training and safety equipment were seen as modifiable factors,…

Treatment of severe hemophilia A with Eloctate and hemophilia B with Alprolix was safe and improved the patients’ annualized bleed rates (ABRs) over four years, according to the results of two extension studies announced by Bioverativ and Sobi. The two open-label extension studies — ASPIRE (NCT01454739), and B-YOND (NCT01425723) — evaluated…