Research graph
References from Real‐World Experience of Mitapivat in Pyruvate Kinase Deficiency. Local targets link to admitted publications; unresolved targets remain external evidence.
Clinical Spectrum of Pyruvate Kinase Deficiency: Data From the Pyruvate Kinase Deficiency Natural History Study
10.1182/blood-2017-10-810796 · 2018 · External reference
The Variable Manifestations of Disease in Pyruvate Kinase Deficiency and Their Management
10.3324/haematol.2019.240846 · 2020 · External reference
Management of Pyruvate Kinase Deficiency in Children and Adults
10.1182/blood.2019000945 · 2020 · External reference
Diagnosis and Management of Pyruvate Kinase Deficiency: International Expert Guidelines
10.1016/s2352-3026(23)00377-0 · 2024 · External reference
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
10.1056/nejmoa1902678 · 2019 · External reference
Mitapivat Versus Placebo for Pyruvate Kinase Deficiency
10.1056/nejmoa2116634 · 2022 · External 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 · 2022 · External reference
Genotype‐Phenotype Correlation and Molecular Heterogeneity in Pyruvate Kinase Deficiency
10.1002/ajh.25753 · 2020 · External 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 · 2021 · External reference
Mitapivat Improves Ineffective Erythropoiesis and Iron Overload in Adult Patients With Pyruvate Kinase Deficiency
10.1182/bloodadvances.2023011743 · 2024 · External reference
Pyruvate Kinase L/R Is a Regulator of Lipid Metabolism and Mitochondrial Function
10.1016/j.ymben.2019.01.001 · 2019 · External reference
Genotype‐Phenotype Correlation and Molecular Heterogeneity in Pyruvate Kinase Deficiency
10.1002/ajh.25753 · ExternalCitation · doi-reference
Pyruvate Kinase L/R Is a Regulator of Lipid Metabolism and Mitochondrial Function
10.1016/j.ymben.2019.01.001 · ExternalCitation · 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 · ExternalCitation · doi-reference
Diagnosis and Management of Pyruvate Kinase Deficiency: International Expert Guidelines
10.1016/s2352-3026(23)00377-0 · ExternalCitation · doi-reference
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
10.1056/nejmoa1902678 · ExternalCitation · doi-reference
Mitapivat Versus Placebo for Pyruvate Kinase Deficiency
10.1056/nejmoa2116634 · ExternalCitation · doi-reference
Clinical Spectrum of Pyruvate Kinase Deficiency: Data From the Pyruvate Kinase Deficiency Natural History Study
10.1182/blood-2017-10-810796 · ExternalCitation · doi-reference
Management of Pyruvate Kinase Deficiency in Children and Adults
10.1182/blood.2019000945 · ExternalCitation · doi-reference
Mitapivat Improves Ineffective Erythropoiesis and Iron Overload in Adult Patients With Pyruvate Kinase Deficiency
10.1182/bloodadvances.2023011743 · ExternalCitation · 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 · ExternalCitation · doi-reference
The Variable Manifestations of Disease in Pyruvate Kinase Deficiency and Their Management
10.3324/haematol.2019.240846 · ExternalCitation · doi-reference
