Internal medicine

Pathology

Placebo

Disease

Gastroenterology

Surgery

Randomized controlled trial

Alternative medicine

Anemia

Sickle cell anemia

Hemoglobin

Confidence interval

Medicine

New England Journal of Medicine

Deoxygenated sickle hemoglobin (HbS) polymerization drives the pathophysiology of sickle cell disease. Therefore, direct inhibition of HbS polymerization has potential to favorably modify disease outcomes. Voxelotor is an HbS polymerization inhibitor.

A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease