News

The U.S. Centers for Disease Control and Prevention (CDC) is seeking input from people who live with pain, as well as their caregivers and healthcare providers to better understand values and preferences related to pain management. This information will help the CDC update and expand the…

Treatment with Idelvion (rIX-FP) helps children and adults with hemophilia B stick to prophylactic dosing schedules by reducing treatment burden, according to a study using clinical trial and real-world data. The study, “High adherence to prophylaxis regimens in haemophilia B patients receiving rIX‐FP: Evidence from clinical…

Long-term prophylaxis, or preventive treatment with Adynovate continues to safely reduce bleeding events over multiple years in children and adults with severe hemophilia A without causing neutralizing antibodies, a Phase 3b trial found. The study, “Long‐term safety and efficacy results from the phase…

Starting factor VIII (FVIII) preventive therapy before the age of 2.5 years improves joint outcomes in young adults with hemophilia A compared to starting treatment later in childhood, a clinical trial shows. The study, “ Young adult outcomes of childhood prophylaxis for severe hemophilia A:…

The U.S. Food and Drug Administration (FDA) has cleared for use CRYOcheck Chromogenic Factor VIII, Precision BioLogic‘s lab test to monitor factor VIII activity in the blood of hemophilia A patients ages 2 and older and help in disease management, the company said in a…

Novalis Biotechnology is supporting a €1.4 million ($1.64 million) investment in Enzyre to finalize the development of a handheld diagnostic device to monitor blood coagulation status in people with hemophilia, and to secure regulatory approval. The funding comes as a Corona Bridge Loan, granted by the…

Adults with mild and moderate hemophilia A have a substantial number of bleeds per year as well as joint problems and surgeries, revealing an unmet medical need in these patients, according to an interim analysis of the CHESS II study. The findings were shared at…

A single dose of the investigational gene therapy BAY 2599023 safely promotes a sustained production of factor VIII (FVIII),  effectively preventing spontaneous bleeds in people with severe hemophilia A, a Phase 1/2 trial shows. Two of the six patients treated to date in this clinical study, both given the…

Treatment with Eloctate (efmoroctocog alfa) was well-tolerated and effective at preventing and treating bleeding episodes in children with severe hemophilia A who had never received any form of therapy for their disease, a Phase 3 trial shows. The study had enrolled boys with severe hemophilia A younger than…

A single infusion of valoctocogene roxaparvovec, BioMarin Pharmaceutical’s investigational gene therapy, can lead to the sustained production of factor VIII (FVIII) in people with hemophilia A, according to a biopsy study that showed the presence of the treatment’s gene constructs in the liver over nearly four…