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Privacy-aware community infrastructureGiacinto Luca Pedone, Paolo Ricchi, Michela Ribersani, Francesco Arcioni, Teresa Ceglie, Filippo Mazzi, Antonella Sau, Maria Pia Solfrizzi, Caterina Giovanna Valentini, Anna Paola Marcello, Paola Ilaria Bianchi, Marta Bortolotti, Francesco Passamonti, Wilma Barcellini, Bruno Fattizzo
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Clinical Spectrum of Pyruvate Kinase Deficiency: Data From the Pyruvate Kinase Deficiency Natural History Study
10.1182/blood-2017-10-810796 · 2018
The Variable Manifestations of Disease in Pyruvate Kinase Deficiency and Their Management
10.3324/haematol.2019.240846 · 2020
Management of Pyruvate Kinase Deficiency in Children and Adults
10.1182/blood.2019000945 · 2020
Diagnosis and Management of Pyruvate Kinase Deficiency: International Expert Guidelines
10.1016/s2352-3026(23)00377-0 · 2024
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
10.1056/nejmoa1902678 · 2019
Mitapivat Versus Placebo for Pyruvate Kinase Deficiency
10.1056/nejmoa2116634 · 2022
Mitapivat in Adult Patients With Pyruvate Kinase Deficiency Receiving Regular Transfusions (ACTIVATE‐T): A Multicentre, Open‐Label, Single‐Arm, Phase 3 Trial
10.1016/s2352-3026(22)00214-9 · 2022
Genotype‐Phenotype Correlation and Molecular Heterogeneity in Pyruvate Kinase Deficiency
10.1002/ajh.25753 · 2020
AG‐348 (Mitapivat), an Allosteric Activator of Red Blood Cell Pyruvate Kinase, Increases Enzymatic Activity, Protein Stability, and ATP Levels Over a Broad Range of PKLR Genotypes
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10.3324/haematol.2019.238865 · 2021
Mitapivat Improves Ineffective Erythropoiesis and Iron Overload in Adult Patients With Pyruvate Kinase Deficiency
10.1182/bloodadvances.2023011743 · 2024
Pyruvate Kinase L/R Is a Regulator of Lipid Metabolism and Mitochondrial Function
10.1016/j.ymben.2019.01.001 · 2019
Pyruvate Kinase L/R Is a Regulator of Lipid Metabolism and Mitochondrial Function
10.1016/j.ymben.2019.01.001 · doi-reference
Mitapivat Improves Ineffective Erythropoiesis and Iron Overload in Adult Patients With Pyruvate Kinase Deficiency
10.1182/bloodadvances.2023011743 · doi-reference
AG‐348 (Mitapivat), an Allosteric Activator of Red Blood Cell Pyruvate Kinase, Increases Enzymatic Activity, Protein Stability, and ATP Levels Over a Broad Range of PKLR Genotypes
10.3324/haematol.2019.238865 · doi-reference
Genotype‐Phenotype Correlation and Molecular Heterogeneity in Pyruvate Kinase Deficiency
10.1002/ajh.25753 · doi-reference
Mitapivat in Adult Patients With Pyruvate Kinase Deficiency Receiving Regular Transfusions (ACTIVATE‐T): A Multicentre, Open‐Label, Single‐Arm, Phase 3 Trial
10.1016/s2352-3026(22)00214-9 · doi-reference
Mitapivat Versus Placebo for Pyruvate Kinase Deficiency
10.1056/nejmoa2116634 · doi-reference
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
10.1056/nejmoa1902678 · doi-reference
Diagnosis and Management of Pyruvate Kinase Deficiency: International Expert Guidelines
10.1016/s2352-3026(23)00377-0 · doi-reference
Management of Pyruvate Kinase Deficiency in Children and Adults
10.1182/blood.2019000945 · doi-reference
The Variable Manifestations of Disease in Pyruvate Kinase Deficiency and Their Management
10.3324/haematol.2019.240846 · doi-reference
Clinical Spectrum of Pyruvate Kinase Deficiency: Data From the Pyruvate Kinase Deficiency Natural History Study
10.1182/blood-2017-10-810796 · doi-reference