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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...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2022
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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 |
Sumario: | 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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