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Bioengineered Factor VIII -- More Innovation for Hemophilia A.
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- Author(s): Chowdary, Pratima
- Source:
New England Journal of Medicine. 7/18/2024, Vol. 391 Issue 3, p277-282. 6p.
- Additional Information
- Subject Terms:
- Abstract:
The article explores the advancements in the treatment of hemophilia A, a bleeding disorder caused by a deficiency of coagulation factor VIII. It focuses on a bioengineered factor VIII recombinant protein called efanesoctocog alfa, which has shown promising results in achieving once-weekly prophylaxis in children with sustained factor VIII levels. The article emphasizes the significance of prolonging the half-life of factor VIII through techniques like endothelial recycling and post-translational modifications. It also discusses the development of a new treatment called efanesoctocog alfa, which has an extended half-life and has been effective in reducing bleeding episodes in patients with previous exposure to factor VIII and no inhibitors. The article acknowledges the importance of conducting pediatric studies for rare diseases and the challenges associated with it. [Extracted from the article]
- Abstract:
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