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Pyruvate kinase activator: A major breakthrough in the world of Hematology

Pyruvate Kinase Deficiency (PKD) is a rare genetic disorder targeting Red Blood Cells that manifests as non-spherocytic hemolytic anemia. It has a global distribution with an unknown prevalence, and the frequently reported estimates for different geographical regions show a significant disparity. Be...

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Detalles Bibliográficos
Autores principales: Iqbal, Amna, Habiba, Ume, Waseem, Radeyah, Islam, Zarmina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9577647/
https://www.ncbi.nlm.nih.gov/pubmed/36268365
http://dx.doi.org/10.1016/j.amsu.2022.104631
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author Iqbal, Amna
Habiba, Ume
Waseem, Radeyah
Islam, Zarmina
author_facet Iqbal, Amna
Habiba, Ume
Waseem, Radeyah
Islam, Zarmina
author_sort Iqbal, Amna
collection PubMed
description Pyruvate Kinase Deficiency (PKD) is a rare genetic disorder targeting Red Blood Cells that manifests as non-spherocytic hemolytic anemia. It has a global distribution with an unknown prevalence, and the frequently reported estimates for different geographical regions show a significant disparity. Because of its hereditary origin, treatment focuses on symptom relief and comfort (principally through blood transfusions, splenectomy, and folic acid supplementation). Pyrukynd (Mitavipat) is a new disease-modifying therapy that was just approved by the FDA and EHA based on clinical trial results that showed a big drop in the number of blood transfusions needed and a rise in hemoglobin levels. The drug stimulates cellular ATP synthesis by acting like Tyrosine Kinase Activator. Even though Pyrukynd has been the subject of studies and is approved for treatment, there is a lack of information on the effects of the medicine on nursing and pregnant mothers. The drug's administration and its effects on minors should also be pleaded.
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spelling pubmed-95776472022-10-19 Pyruvate kinase activator: A major breakthrough in the world of Hematology Iqbal, Amna Habiba, Ume Waseem, Radeyah Islam, Zarmina Ann Med Surg (Lond) Short Communication Pyruvate Kinase Deficiency (PKD) is a rare genetic disorder targeting Red Blood Cells that manifests as non-spherocytic hemolytic anemia. It has a global distribution with an unknown prevalence, and the frequently reported estimates for different geographical regions show a significant disparity. Because of its hereditary origin, treatment focuses on symptom relief and comfort (principally through blood transfusions, splenectomy, and folic acid supplementation). Pyrukynd (Mitavipat) is a new disease-modifying therapy that was just approved by the FDA and EHA based on clinical trial results that showed a big drop in the number of blood transfusions needed and a rise in hemoglobin levels. The drug stimulates cellular ATP synthesis by acting like Tyrosine Kinase Activator. Even though Pyrukynd has been the subject of studies and is approved for treatment, there is a lack of information on the effects of the medicine on nursing and pregnant mothers. The drug's administration and its effects on minors should also be pleaded. Elsevier 2022-09-14 /pmc/articles/PMC9577647/ /pubmed/36268365 http://dx.doi.org/10.1016/j.amsu.2022.104631 Text en © 2022 The Authors https://creativecommons.org/licenses/by/4.0/This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Short Communication
Iqbal, Amna
Habiba, Ume
Waseem, Radeyah
Islam, Zarmina
Pyruvate kinase activator: A major breakthrough in the world of Hematology
title Pyruvate kinase activator: A major breakthrough in the world of Hematology
title_full Pyruvate kinase activator: A major breakthrough in the world of Hematology
title_fullStr Pyruvate kinase activator: A major breakthrough in the world of Hematology
title_full_unstemmed Pyruvate kinase activator: A major breakthrough in the world of Hematology
title_short Pyruvate kinase activator: A major breakthrough in the world of Hematology
title_sort pyruvate kinase activator: a major breakthrough in the world of hematology
topic Short Communication
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9577647/
https://www.ncbi.nlm.nih.gov/pubmed/36268365
http://dx.doi.org/10.1016/j.amsu.2022.104631
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