ENERGIZE: A GLOBAL PHASE 3 STUDY OF MITAPIVAT DEMONSTRATING EFFICACY AND SAFETY IN ADULTS WITH ALPHA- OR BETA-NON–TRANSFUSION-DEPENDENT THALASSEMIA

ENERGIZE: A GLOBAL PHASE 3 STUDY OF MITAPIVAT DEMONSTRATING EFFICACY AND SAFETY IN ADULTS WITH ALPHA- OR BETA-NON–TRANSFUSION-DEPENDENT THALASSEMIA

Background: In thalassemia, ATP production in erythroid cells is too low to meet the demand of oxidative stress and ensuing cellular damage; this leads to ineffective erythropoiesis (IE) and chronic hemolytic anemia. Guidelines for non–transfusion-dependent thalassemia (NTDT) recommend raising hemog...

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Main Authors: AT Taher, H Al-Samkari, Y Aydinok, M Besser, G Luna, S Gheuens, A Glenthj, AS Goh, A Kattamis, SR Loggetto, KM Musallam, P Ricchi, E Salido-Fiérrez, S Sheth, V Viprakasit, MD Cappellini, KH Kuo
Format: Article
Language:English
Published: Elsevier 2024-10-01
Series:Hematology, Transfusion and Cell Therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2531137924003882
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