Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia—TCD With Transfusions Changing to Hydroxyurea (TWiTCH): a multicentre, open-label, phase 3, non-inferiority trialRussell E. Ware, Robert J. Adams, Barry R. Davis et al.|The Lancet|2015Cited by 518
Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History StudyRachael F. Grace, Wilma Barcellini, Paola Bianchi et al.|Blood|2018Cited by 165
Safety and Efficacy of Mitapivat in Pyruvate Kinase DeficiencyRachael F. Grace, Bertil Glader, Christian Rosé et al.|New England Journal of Medicine|2019Cited by 162
Genotype‐phenotype correlation and molecular heterogeneity in pyruvate kinase deficiencyPaola Bianchi, Rachael F. Grace, Kevin H.M. Kuo et al.|American Journal of Hematology|2020Cited by 66
Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate Kinase Deficiency Natural History StudyEduard J. van Beers, Rachael F. Grace, Stephanie van Straaten et al.|Haematologica|2018Cited by 61