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Biology & Medicine · 23/10/2026 20:08 UTC

[Comment] Mitapivat in the RISE UP trial: a new therapy for sickle cell disease

Sickle cell disease is the most common haemoglobinopathy, affecting more than 7 million people worldwide.1 Despite substantial morbidity and mortality, access to…

Sickle cell disease is the most common haemoglobinopathy, affecting more than 7 million people worldwide.1 Despite substantial morbidity and mortality, access to effective disease-modifying therapies remains limited.2,3 Hydroxyurea and chronic blood transfusions have both been shown to reduce acute sickle cell disease complications, including vaso-occlusive crises and stroke, while also improving haematological parameters.4,5 However, the availability and long-term feasibility of both could be l

Source: The Lancet

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