Hemophilia News Today brought you daily coverage of important discoveries, treatment developments, clinical trials, and other key events related to hemophilia throughout 2019. As we look forward to bringing you more news in 2020, we would like to remind you of the 10 most-read stories of 2019. No.
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The full range of hemophilia treatments should be made available to patients without consideration of cost or other barriers, leaders of patient associations and others argued at a public meeting hosted by the Institute for Clinical and Economic Review (ICER) to consider its recent analysis of hemophilia A therapies. The…
Hemophilia B patients living in urban mainland China have longer hospitalizations and significantly higher medical costs — including more expensive fees for coagulation factors concentrates — compared with patients with hemophilia A, a retrospective study shows. In fact, people with hemophilia B had inpatient medical costs more than…
Preventive treatment with Kovaltry (octocog alfa) is safe and effective at preventing bleeds, including joint bleeds, for up to two years in people with hemophilia A, a long-term extension study shows. Findings were reported in the study “BAY 81-8973 demonstrated efficacy, safety and joint status improvement…
Clinical trials of Hemlibra (emicizumab-kxwh) continue to show significant reductions in bleeding episodes in patients with hemophilia A with inhibitors, Genentech recently announced. Data from Hemlibra’s clinical development program were presented at the 59th American Society of Hematology (ASH) Annual Meeting. The results show that in hemophilia A patients with inhibitors…
Investigational gene therapy SB-525 can safely induce durable clotting factor VIII (FVIII) activity in patients with severe hemophilia A, preliminary data from the Phase 1/2 Alta clinical trial show. The trial’s most recent results were discussed at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress in…
Long-term prophylactic (preventive) treatment with a fixed dose of Esperoct (turoctocog alfa pegol) — approved in the U.S. and the EU in 2019 — was effective in preventing and stopping bleeding in people with severe hemophilia A, according to data from two Phase 3 clinical trials. Findings from…
Cells derived from hemophilia A patients — genetically reprogrammed to produce a functional clotting factor VIII (FVIII) that’s missing or not working in people with the blood disease — were successfully grafted into hemophiliac mice, restoring blood levels of FVIII and significantly improving…
In the U.S., hemophilia A is estimated to affect one in every 5,000 male births, hemophilia B one in 25,000 male births, and hemophilia C one in every 100,000 people (males and females). Hemophilia A is thus four times more common than hemophilia B, and about 10 times more common than…
Hemophilia News Today brought you daily coverage in 2020 of important discoveries, treatment advancements, clinical trial findings, and other important events related to hemophilia. As we look forward to bringing you more news this year, we present here the 10 most-read stories of 2020, along with a summary of…