1.
Kuppens GZ, Ruiz MAD, van Dijk MJ, Rab MA, Derichs C, Saes J, Rijneveld AW, Cnossen MH, Nur E, Biemond BJ, Bartels M, Saraf SL, van Beers EJ. Three-year safety, efficacy, and renal outcomes of mitapivat treatment in sickle cell disease: results from the phase 2 open-label study. Haematologica; https://doi.org/10.3324/haematol.2025.289105 [Early view].