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10 p, 489.9 KB The Pyruvate Kinase Deficiency Global Longitudinal (Peak) Registry : rationale and study design / Grace, Rachael F. (Dana-Farber/Boston Children's Cancer and Blood Disorders Center) ; Van Beers, Eduard J. (University of Utrecht) ; Vives Corrons, Joan Lluís (Institut Germans Trias i Pujol. Institut de Recerca contra la Leucèmia Josep Carreras) ; Glader, Bertil (Stanford University School of Medicine) ; Glenthøj, Andreas (Copenhagen University Hospital - Rigshospitalet) ; Kanno, Hitoshi (Department of Transfusion Medicine and Cell Processing. Tokyo Women's Medical University) ; Kuo, Kevin H M (University of Toronto) ; Lander, Carl (Hammersmith Hospital (Londres)) ; Pospíšilová, Dagmar (Department of Pediatrics. Palacky University and University Hospital) ; Viprakasit, Vip (Mahidol University) ; Li, Junlong (Agios Pharmaceuticals) ; Yan, Yan (Agios Pharmaceuticals (Estats Units d'Amèrica)) ; Boscoe, Audra N. (Agios Pharmaceuticals (Estats Units d'Amèrica)) ; Bianchi, P. (Fondazione Irccs Ca' Granda Ospedale Maggiore Policlinico (Itàlia))
Introduction Pyruvate kinase (PK) deficiency is a rare, under-recognised, hereditary condition that leads to chronic haemolytic anaemia and potentially serious secondary complications, such as iron overload, cholecystitis, pulmonary hypertension and extramedullary haematopoiesis. [...]
2023 - 10.1136/bmjopen-2022-063605
BMJ open, Vol. 13 Núm. 3 (23 2023) , p. e063605  
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2 Kanno, H.
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