Myrthe J van Dijk, Minke A E Rab, Brigitte A van Oirschot, Jennifer Bos, Cleo Derichs, Anita W Rijneveld, Marjon H Cnossen, Erfan Nur, Bart J Biemond, Marije Bartels, Judith J M Jans, W W van Solinge, Roger E G Schutgens, Richard van Wijk, Eduard J van Beers
Targeting the primary pathogenic event of sickle cell disease (SCD), polymerization of sickle hemoglobin (HbS), may prevent downstream clinical events. Mitapivat, an oral pyruvate kinase (PK) activator, has therapeutic potential by increasing adenosine triphosphate (ATP) and decreasing 2,3-diphosphoglycerate (2,3-DPG), a glycolytic red blood cell (RBC) intermediate. In the previously reported 8-week dose-finding period of this phase 2, investigator initiated, open-label study, mitapivat was well tolerated and showed efficacy in SCD...
November 7, 2023: Blood Advances