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Hemophilia A is a bleeding disorder caused by F8 gene mutations, leading to insufficient factor VIII. Treatment options include long-term preventive use of Esperoct and Hemlibra, which received breakthrough therapy designation for hemophilia A without factor VIII inhibitors. Patients may develop inhibitors to factor VIII, with intensive treatment increasing this risk in non-severe cases. Support groups offer resources and emotional support for managing the condition.
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The U.S. Food and Drug Administration granted breakthrough therapy designation to Genentech’s Hemlibra (emicizumab-kxwh) for people with hemophilia A without factor VIII inhibitors. Hemlibra is designed to bring together the proteins required to activate the natural coagulation cascade and restore the blood clotting process for patients with…
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…
Hemophilia A patients who receive replacement therapies of factor VIII often develop inhibitors against factor concentrates which hinders the effectiveness of the therapy, increases disability, and reduces quality of life. Although immune tolerance induction (ITI) can reduce inhibitors against factor VIII concentrates, the study “Long-term course of…
Intensive treatment with factor VIII, the clotting protein missing or defective in patients with hemophilia A, is associated with a greater risk of inhibitor development among patients with non-severe disease, a study has found. The findings highlight the importance of closely monitoring these patients in order to prevent or…
A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and, in 16 of 18 male patients, those levels were sustained for up to four years, according to results from a Phase 1/2 clinical…
Social Clips
How to Explain Hemophilia to Children
In this simple animated video from CSL Behring US, hemophilia is explained in easy-to-understand language aimed at children and caregivers of newly diagnosed hemophilia patients. MORE: Explaining hemophilia A and hemophilia B The narrator shares the basic difference between hemophilia A and hemophilia B, and that…
Precision BioLogic revealed promising data on a new kit for the measurement of FVIII inhibitors (factor VIII) in patients with hemophilia A. The results were presented at the International Society on Thrombosis and Haemostasis’ Scientific and Standardization Committee (SSC) meeting July 18-21 in Dublin, Ireland. Researchers presented their…
News
Advantages, Unanswered Questions About PEGylated Products for Hemophilia A Reviewed by Researchers
Researchers reviewed the current hemophilia research that focuses on the development of improved longer-acting factor replacement therapies, particularly PEGylated products. The new clinical research advances lead the scientific community to look at such developments with optimism, but also some caution. The study, “Potential role of a new PEGylated recombinant…