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Am J Hematol. 2012 May;87 Suppl 1:S33-9. doi: 10.1002/ajh.23146. Epub 2012 Mar 3.

The hope and reality of long-acting hemophilia products.

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Department of Pediatrics, University of Michigan Medical Center, Ann Arbor, Michigan 48109, USA.


Recombinant DNA technology and protein engineering are creating hope that we can address ongoing challenges in hemophilia care such as reducing the costs of therapy, increasing the availability to the developing world, and improving the functional properties of these proteins. Technological advances to improve the half-life of recombinant clotting factors have brought long-acting clotting factors for hemophilia replacement therapy closer to reality. Preclinical and clinical trial results are reviewed as well as the potential benefits and risks of these novel therapies.

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