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Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in sickle cell disease: A phase 2, open-label study.

AuthorsMyrthe 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, Wouter W van Solinge, Roger E G Schutgens, Richard van Wijk, Eduard J van Beers
JournalAmerican journal of hematology (Am J Hematol) Vol. 97 Issue 7 Pg. E226-E229 (07 2022) ISSN: 1096-8652 [Electronic] United States
PMID35384026 (Publication Type: Clinical Trial, Phase II, Letter, Research Support, Non-U.S. Gov't)
Chemical References
  • Piperazines
  • Quinolines
  • mitapivat
  • Pyruvate Kinase
Topics
  • Anemia, Sickle Cell (drug therapy)
  • Humans
  • Piperazines (adverse effects)
  • Pyruvate Kinase
  • Quinolines (adverse effects)
  • Treatment Outcome

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