Patricia Inácio, PhD, science writer —

After 18 months, the investigational gene therapy AMT-061 (etranacogene dezaparvovec) continued to prevent bleeds in men with moderate-to-severe hemophilia B, according to top-line data from the Phase 3 HOPE-B clinical trial. The trial has met its pre-specified primary goal, with control of all bleeds by 18 months not…

BioMatrix Specialty Pharmacy has announced this year’s winners of its memorial scholarship program, designed to help people with hemophilia and other bleeding disorders pursue higher education. Six scholarships, each worth $1,000 and honoring people who made “unique” contributions to the bleeding disorder community during their life, are awarded…

Chronic pain and a reliance on pain killers remains high among adult men with severe hemophilia B after two years on preventive treatment with extended half-life recombinant factor IX — even with a lesser bleeding frequency and fewer repeated joint bleeds, a real-world study from Ireland reports. These findings…

Hemophilia patients experienced improvements in joint health, mobility, and overall quality of life after completing a supervised eight-week exercise program, a study reports. However, in order to achieve long-lasting benefits, regular exercise may be required. The study, “Effects of a supervised therapeutic exercise program on musculoskeletal health and…

Preventive treatment with factor VIII (FVIII) — the missing blood clotting protein in hemophilia A — was not fully effective at preventing MRI-detected joint changes in boys with severe disease, according to a Canadian study. Regular evaluation of joints by MRI or ultrasounds, however, may help in identifying those…

Eptacog beta was safe and 100% effective at preventing bleeds during and after minor surgeries in hemophilia A patients with inhibitors, according to data from a Phase 3 trial. The treatment’s efficacy at managing bleeds dropped with major surgeries, with post-operative success seen in 66.7% of evaluated cases. Overall, these…

A report describes — for the first time, according to study authors — the case of a girl who has both hemophilia A and C. To date, only 10 such cases had been documented in medical literature, all occurring in men. The case report authors highlight the importance of…

Belief Biomed is launching a Phase 1/2 clinical trial in China to test BBM-H901, its investigational one-time gene therapy for hemophilia B. The announcement comes after China’s National Medical Products Administration (NMPA) approved the company’s investigational new drug application, which had been submitted in April, Belief Biomed said in…

In the Netherlands, children and adults with hemophilia participate in sports as much or more than people from the general population, according to a study. The findings also indicated that disease severity affected sports participation only in adults, and that children with hemophilia were more willing to participate in…

The European Medicines Agency (EMA) has validated BioMarin Pharmaceutical’s new application seeking approval of its investigational one-time gene therapy Roctavian (valoctocogene roxaparvovec) for severe hemophilia A. The application was resubmitted to EMA following a request for longer follow-up data from the Phase 3 GENEr8-1 clinical trial (NCT03370913),…