Mitapivat versus Placebo for Pyruvate Kinase Deficiency

N Engl J Med. 2022 Jun 30;386(26):2538-2539. doi: 10.1056/NEJMc2206275.
No abstract available

Publication types

  • Letter
  • Comment

MeSH terms

  • Anemia, Hemolytic, Congenital Nonspherocytic*
  • Humans
  • Piperazines
  • Pyruvate Kinase / deficiency
  • Pyruvate Metabolism, Inborn Errors*
  • Quinolines

Substances

  • Piperazines
  • Quinolines
  • mitapivat
  • Pyruvate Kinase

Supplementary concepts

  • Pyruvate Kinase Deficiency of Red Cells