Health Canada has approved Hemlibra (emicizumab) for people with Hemophilia A without factor VIII inhibitors. Hemlibra is an antibody therapy that works by combining factors IX and X of the blood clotting cascade, effectively “replacing” the function of factor VIII, which is lacking in Hemophilia A patients. It is…
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People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…
High adherence to prophylactic treatment not only reduces the number of spontaneous bleeding episodes in patients with hemophilia A, but it also improves their physical activity, according to a study. The study, “Physical activity improved by adherence to prophylaxis in an Italian population of children, adolescents and…
The emotional toll of the “tainted blood era” is still vivid in the minds of healthcare professionals who felt helpless to stop the spread of epidemics among patients receiving treatment for blood disorders in Canada during the 1980s. Learning from these mistakes and developing support programs to help clinicians overcome…
The vast majority of people in the hemophilia community support the implementation of newborn screening for the bleeding disorder in the United Kingdom, a study reports. The study, “Newborn screening for haemophilia: The views of families and adults living with haemophilia in the UK,” was published in…
More than half of people with hemophilia in Japan fail to take part in sports or other physical activities at levels thought necessary to maintaining good health, a study reports. New strategies for education, support, and guidance are needed to promote better physical activity among this population, it recommended The…
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…
Treatment with Esperoct led to stabilization and/or improvement of health-related quality of life (HRQoL) in patients with severe hemophilia A from all age groups, a study suggests. The study, “Health-related quality-of-life and treatment satisfaction of individuals with hemophilia A treated with turoctocog alfa pegol (N8-GP): a new recombinant…
BioMarin Pharmaceutical’s investigative gene therapy, called valoctocogene roxaparvovec, is capable of sustained control of bleeding rate requiring factor VIII infusions by at least 92% in adults with severe hemophilia A, three-year data from a Phase 1/2 trial show. Moreover, a model predicts that the…
A new five-year alliance between Bayer and the World Federation of Hemophilia (WFH) Humanitarian Aid Program will deliver training, education and treatments to healthcare professionals in more than 60 underserved countries. ”Three out of four people with bleeding disorders living in developing countries do not have access to…
