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A multicenter, phase III, double-blind, randomized, placebo-controlled trial in persons with sickle cell disease.
Primary outcome measures:
Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24
Conclusion:
Voxelotor increased hemoglobin levels and reduced markers of hemolysis. These findings align with HbS polymerization inhibition. They show disease-modifying potential.
Vichinsky et al. 2019 N Engl J Med 381: 509-519
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