A Phase IIa, Double Blind, Randomized, Placebo Controlled, Parallel, Multicentre, Proof-of-Concept Study to Evaluate the Efficacy and Safety of Desidustat Oral Tablet for Treatment of Sickle Cell Disease.
INTRODUCTION Sickle cell disease (SCD) is an inherited hemoglobin disorder caused by a point mutation in the β-globin gene, producing hemoglobin S (HbS). Under hypoxic conditions, HbS polymerizes, leading to rigid, sickled erythrocytes with reduced deformability and increased endothelial adhesion, resulting in vaso-occ...