A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…
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The ongoing Phase 1/2 Alta trial continues to show that a single infusion of SB-525, an investigational gene therapy, leads to stable and clinically relevant improvements in clotting factor VIII and marked reductions in the need for replacement therapy in patients with hemophilia A. This preliminary follow-up data…
Self-reported health outcomes of hemophilia patients provide useful and clinically valuable data on the efficacy of treatments, a population-based study shows. The study, “Real-world utilities and health-related quality-of-life data in hemophilia patients in France and the United Kingdom,” was published in…
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…
For the second year, key pharmaceutical and academic leaders in hemophilia therapy development will gather at the Hemophilia Drug Development Summit — to be held in Boston Aug. 20–22 —to discuss and advance the next generation of safe and more effective therapies for bleeding disorders. The recent approval of Genentech’s …
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…
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…
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High Adherence to Prophylaxis Improves Physical Activity of Patients With Hemophilia A, Study Says
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…
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