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Right in time: Mitapivat for the treatment of anemia in α- and β-thalassemia
Kuo and colleagues(1) evaluated the safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion-dependent α-thalassemia or β-thalassemia. The high rate of hemoglobin response and good tolerability encourages further development in thalassemia.
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/PMC9589095/ https://www.ncbi.nlm.nih.gov/pubmed/36260990 http://dx.doi.org/10.1016/j.xcrm.2022.100790 |
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author | Musallam, Khaled M. Taher, Ali T. Cappellini, Maria Domenica |
author_facet | Musallam, Khaled M. Taher, Ali T. Cappellini, Maria Domenica |
author_sort | Musallam, Khaled M. |
collection | PubMed |
description | Kuo and colleagues(1) evaluated the safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion-dependent α-thalassemia or β-thalassemia. The high rate of hemoglobin response and good tolerability encourages further development in thalassemia. |
format | Online Article Text |
id | pubmed-9589095 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | Elsevier |
record_format | MEDLINE/PubMed |
spelling | pubmed-95890952022-10-25 Right in time: Mitapivat for the treatment of anemia in α- and β-thalassemia Musallam, Khaled M. Taher, Ali T. Cappellini, Maria Domenica Cell Rep Med Spotlight Kuo and colleagues(1) evaluated the safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion-dependent α-thalassemia or β-thalassemia. The high rate of hemoglobin response and good tolerability encourages further development in thalassemia. Elsevier 2022-10-18 /pmc/articles/PMC9589095/ /pubmed/36260990 http://dx.doi.org/10.1016/j.xcrm.2022.100790 Text en © 2022 The Authors https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/). |
spellingShingle | Spotlight Musallam, Khaled M. Taher, Ali T. Cappellini, Maria Domenica Right in time: Mitapivat for the treatment of anemia in α- and β-thalassemia |
title | Right in time: Mitapivat for the treatment of anemia in α- and β-thalassemia |
title_full | Right in time: Mitapivat for the treatment of anemia in α- and β-thalassemia |
title_fullStr | Right in time: Mitapivat for the treatment of anemia in α- and β-thalassemia |
title_full_unstemmed | Right in time: Mitapivat for the treatment of anemia in α- and β-thalassemia |
title_short | Right in time: Mitapivat for the treatment of anemia in α- and β-thalassemia |
title_sort | right in time: mitapivat for the treatment of anemia in α- and β-thalassemia |
topic | Spotlight |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9589095/ https://www.ncbi.nlm.nih.gov/pubmed/36260990 http://dx.doi.org/10.1016/j.xcrm.2022.100790 |
work_keys_str_mv | AT musallamkhaledm rightintimemitapivatforthetreatmentofanemiainaandbthalassemia AT taheralit rightintimemitapivatforthetreatmentofanemiainaandbthalassemia AT cappellinimariadomenica rightintimemitapivatforthetreatmentofanemiainaandbthalassemia |