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December 8, 2025Blood

Long-term mitapivat treatment improves inflammatory pro-fibrotic cardiomyopathy in a murine model of β-thalassemia.

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Long-term mitapivat treatment reduces heart inflammation and fibrosis in β-thalassemia, suggesting potential for cardiomyopathy management.

Mitapivat treatment led to significant improvements in inflammatory pro-fibrotic cardiomyopathy in mice with β-thalassemia.
Metabolomic analyses showed elevated sphingosine 1-phosphate and normalization of acyl-carnitine levels in treated mice.
Assessment using lipidomic and proteomic approaches revealed a significant reduction in inflammatory and fibrotic markers post-treatment.
This research highlights the potential of mitapivat for cardiovascular protection in β-thalassemia models, necessitating further investigation.

Riccardi et al. (2025) studied this question.

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Long-term mitapivat treatment reduces heart inflammation and fibrosis in β-thalassemia, suggesting potential for cardiomyopathy management.