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Comment
. 2022 Oct 18;3(10):100790.
doi: 10.1016/j.xcrm.2022.100790.

Right in time: Mitapivat for the treatment of anemia in α- and β-thalassemia

Khaled M Musallam  1 , Ali T Taher  2 , Maria Domenica Cappellini  3
Affiliations
Comment

Right in time: Mitapivat for the treatment of anemia in α- and β-thalassemia

Khaled M Musallam et al. Cell Rep Med. .

Abstract

Kuo and colleagues1 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.

Keywords: anemia; ineffective erythropoiesis; thalassemia.

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Conflict of interest statement

Declaration of interests K.M.M. reports consultancy fees from Novartis, Celgene Corp (Bristol Myers Squibb), Agios Pharmaceuticals, CRISPR Therapeutics, Vifor Pharma, and Pharmacosmos. M.D.C. reports consultancy fees from Novartis, Celgene Corp (Bristol Myers Squibb), Vifor Pharma, and Ionis Pharmaceuticals and research funding from Novartis, Celgene Corp (Bristol Myers Squibb), La Jolla Pharmaceutical Company, Roche, Protagonist Therapeutics, and CRISPR Therapeutics. A.T.T. reports consultancy fees from Novartis, Celgene Corp (Bristol Myers Squibb), Vifor Pharma, Silence Therapeutics, and Ionis Pharmaceuticals and research funding from Novartis, Celgene Corp (Bristol Myers Squibb), La Jolla Pharmaceutical Company, Roche, Protagonist Therapeutics, and Agios Pharmaceuticals.

Comment on

References

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