[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 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 limited in some settings.