It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although she had known for years that something was wrong, the news that David would battle a lifelong rare disease devastated…
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:…
Bayer’s Jivi, an FVIII replacement therapy for hemophilia A, has a longer half-life and a slower clearance from blood circulation than Eloctate, and may protect against bleeding for longer periods, results of a Phase 1 trial in people with severe disease suggests. The study “Direct comparison of…
Based on feedback from the hemophilia B community, Aptevo Therapeutics announced that a 3,000 international unit (IU) vial assay of Ixinity is now available for patients preferring the convenience of a larger assay over the smaller IUs on the market for this intravenous recombinant factor IX. The…
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…
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…
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