Emerging drugs in randomized controlled trials for sickle cell disease: are we on the brink of a new era in research and treatment?

Abstract excerpt

Introduction: Sickle cell disease (SCD) is caused by a mutation in the HBB gene which is key for making a component of hemoglobin. The mutation leads to the formation of an abnormal hemoglobin molecule called sickle hemoglobin (HbS). SCD is a chronic, complex disease with a multiplicity of pathophysiological targets; it has high morbidity and mortality.Hydroxyurea has for many years been the only approved drug...

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