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Author

M. Cappellini

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Clinical trial Sep 2026

Efficacy and safety of mitapivat in adults with transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE-T): a double-blind, randomised, multicentre, placebo-controlled, phase 3 trial.

Findings support mitapivat as the first oral disease-modifying therapy for adults with transfusion-dependent α-thalassaemia or β-thalassaemia, providing a new treatment option to reduce transfusion burden in this patient population.

M. Cappellini, Sujit Sheth, A. Taher et al. · 3 citations
#gene editing Review Oct 2026

The expanding therapeutic landscape of β-thalassaemia.

Advances in β-thalassaemia treatment over the past decade are discussed, including newly approved and late-stage therapies and their future implications, and remaining research gaps are highlighted.

K. Musallam, Sujit Sheth, Franco Locatelli et al. · 2 citations
Review Open access Aug 2026

2026 Update on Clinical Trials in β-Thalassemia.

Several promising approaches targeting fetal hemoglobin induction, iron metabolism, and ineffective erythropoiesis have failed to demonstrate sufficient clinical benefit despite preclinical proof of concept, highlighting the complexity of therapeutic development in β-thalassemia.

K. M. Musallam, F. Locatelli, M. Algeri et al. · 0 citations

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