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The Thalassemia Channel is supported with funding from Agios (Gold).
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ESH Erythro 2025 | Promising therapies for SCD & thalassemia: gene therapy, luspatercept, mitapivat & more
In this video, Perla Eleftheriou, MRCP, FRCpath, University College London Hospitals NHS Foundation Trust, London, UK, gives an overview of exciting therapies being explored in sickle cell disease (SCD) and thalassemia, highlighting the recent approval of CRISPR-based gene editing for these diseases and the potential for expanding treatment accessibility. Dr Eleftheriou notes that eligibility remains a challenge for these gene therapies and further discusses the benefits of luspatercept and mitapivat. This interview took place at the 4th ESH Translational Research Conference on Pathophysiology and Clinical Advances in Sickle Cell Disease and Thalassemia in Dublin, Ireland.
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