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 · 2022

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Abstract

Kuo and colleagues<sup>1</sup> 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.

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