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Open access Sep 2026

Efficacy and safety of thalidomide (1 vs 2 mg/kg/d) in transfusion-dependent thalassemia: A Non-Inferiority Trial.

Thalidomide has emerged as a fetal hemoglobin-inducer with potential to reduce transfusion burden in transfusion-dependent thalassemia (TDT). However, optimal dosing remains undefined A prospective, randomized, open-label, multicentric clinical trial was conducted at four centres in India to compare efficacy and safety...

S. Kakkar, Amita Mahajan, R. Sharma et al. · 1 citation
Review Oct 2026

Future-proofing thalassaemia care: harnessing scalable therapies, artificial intelligence, and global collaboration.

The emerging role of repurposed low-cost therapies, particularly hydroxyurea and thalidomide, as disease-modifying options in resource-limited settings are discussed and opportunities to democratise access to curative gene therapies through technological developments, decentralised manufacturing, regional collaboration...

Hanny T. Al-Samkari, F. Aboobacker, B. Andemariam et al. · 2 citations

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