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Browsing by Author "Grace, RF"
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The Burden of Disease in Pyruvate Kinase Deficiency: Patients' Perception of the Impact on Health-Related Quality of Life
Grace, RF; Cohen, L; Egan, S; Wells, T; Witherspoon, B; Ryan, A; Salek, Mir-Saeed Shayegan; Bodie, S; Klaassen, RJ (2018-12-01)Objectives: This study explored how signs and symptoms of pyruvate kinase (PK) deficiency, a rare hemolytic anemia caused by mutations in the PKLR gene, impacts patients’ health-related quality of life (HRQoL). Methods: ... -
Psychometric validation of the Pyruvate Kinase Deficiency Diary and Pyruvate Kinase Deficiency Impact Assessment in adults in the phase 3 ACTIVATE trial
Andrae, David A.; Grace, RF; Jewett, Adrian; Foster, Brandon; Klaassen, Robert J.; Salek, Sam; Li, Junlong; Tai, Feng; Boscoe, Audra N.; Zagadailov, Erin (2023-11-09)Background: Pyruvate kinase (PK) deficiency is a rare hereditary disorder characterized by chronic hemolytic anemia and serious sequalae which negatively affect patient quality of life. This study aimed to psychometrically ...