Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or β-thalassaemia: an open-label …
KHM Kuo, DM Layton, A Lal, H Al-Samkari, J Bhatia… - The Lancet, 2022 - thelancet.com
Background Patients with non-transfusion-dependent thalassaemia (NTDT), although they
do not require regular blood transfusions for survival, can still accrue a heavy burden of …
do not require regular blood transfusions for survival, can still accrue a heavy burden of …
The pyruvate kinase activator mitapivat reduces hemolysis and improves anemia in a β-thalassemia mouse model
Anemia in β-thalassemia is related to ineffective erythropoiesis and reduced red cell
survival. Excess free heme and accumulation of unpaired α-globin chains impose …
survival. Excess free heme and accumulation of unpaired α-globin chains impose …
[HTML][HTML] Erythropoiesis in lower-risk myelodysplastic syndromes and beta-thalassemia
The hematologic disorders myelodysplastic syndromes and beta-thalassemia are
characterized by ineffective erythropoiesis and anemia, often managed with regular blood …
characterized by ineffective erythropoiesis and anemia, often managed with regular blood …
A fit-for-purpose LC–MS/MS method for the simultaneous quantitation of ATP and 2, 3-DPG in human K2EDTA whole blood
H Kim, P Kosinski, C Kung, L Dang, Y Chen… - … of Chromatography B, 2017 - Elsevier
Many hemolytic anemias results in major metabolic abnormalities: two common metabolite
abnormalities include increased levels of 2, 3-diphosphoglycerate (2, 3-DPG) and …
abnormalities include increased levels of 2, 3-diphosphoglycerate (2, 3-DPG) and …
Pyruvate kinase activators: targeting red cell metabolism in thalassemia
KHM Kuo - Hematology, 2023 - ashpublications.org
Thalassemia is an inherited red blood cell disorder whereby the qualitative and/or
quantitative imbalance in α-to β-globin ratio results in hemolysis and ineffective …
quantitative imbalance in α-to β-globin ratio results in hemolysis and ineffective …
Myostatin Knockout Limits Exercise-Induced Reduction in Bovine Erythrocyte Oxidative Stress by Enhancing the Efficiency of the Pentose Phosphate Pathway
Simple Summary Myostatin (MSTN) is mainly expressed in skeletal muscle and is involved
in the regulation of skeletal muscle growth and development. Loss of MSTN results in …
in the regulation of skeletal muscle growth and development. Loss of MSTN results in …
Mitapivat sulfate. Pyruvate kinase isozyme R activator, Treatment of pyruvate kinase deficiency, Treatment of thalassemia, Treatment of sickle cell disease
W Barcellini, JA Giannotta, N Cecchi… - Drugs of the …, 2021 - access.portico.org
Summary Mitapivat (AG-348) is an oral, allosteric activator of pyruvate kinase (PK) that
catalyzes the final step of glycolysis. The drug is active on both wild-type and mutant …
catalyzes the final step of glycolysis. The drug is active on both wild-type and mutant …
[HTML][HTML] Mitapivat (AG-348), an oral PK-R activator, in adults with non-transfusion dependent thalassemia: a phase 2, open-label, multicenter study in progress
Background: Thalassemia is a group of inherited blood disorders in which genetic mutation
(s) in the α-and/or β-globin locus lead to excess precipitation of β-or α-globin, respectively …
(s) in the α-and/or β-globin locus lead to excess precipitation of β-or α-globin, respectively …
[引用][C] Development and validation of a LC-MS/MS method for the simultaneous detection and quantitation of adenosine-5'-triphosphate, 2, 3-bisphosphoglyceric acid …
APDH Oberacher - 2021 - Medical University of Innsbruck