@article{d769bb671261445080f5f95a1172c31c,
title = "Clinically meaningful improvements in patient-reported outcomes in mitapivat-treated patients with pyruvate kinase deficiency",
abstract = "Clinically meaningful benefits in the signs, symptoms, and impacts of #PKDeficiency as assessed by disease-specific patient-reported outcome measures were observed in mitapivat-treated adult patients in two phase 3 clinical trials.",
keywords = "Adult, Anemia, Hemolytic, Congenital Nonspherocytic, Female, Humans, Male, Middle Aged, Patient Reported Outcome Measures, Pyruvate Kinase/deficiency, Pyruvate Metabolism, Inborn Errors, Treatment Outcome",
author = "Kuo, {Kevin H M} and Grace, {Rachael F} and {van Beers}, {Eduard J} and Wilma Barcellini and Andreas Glenth{\o}j and Susanne Holzhauer and Vanessa Beynon and Susan Morris and Junlong Li and Erin Zagadailov and Parija Patel and Hanny Al-Samkari",
note = "{\textcopyright} 2024 The Authors. American Journal of Hematology published by Wiley Periodicals LLC.",
year = "2024",
doi = "10.1002/ajh.27312",
language = "English",
volume = "99",
pages = "1415--1419",
journal = "American Journal of Hematology",
issn = "0361-8609",
publisher = "John Wiley and Sons Inc.",
number = "7",
}