A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease

Vichinsky, Elliott; Hoppe, Carolyn; I. Ataga, Kenneth; E. Ware, Russell; Nduba, Videlis; El‐Beshlawy, Amal; Hassab, Hoda; M. Achebe, Maureen; Alkindi, Salam; Clark Brown, R.; Diuguid, David; Telfer, Paul; A. Tsitsikas, Dimitris; Elghandour, Ashraf; R. Gordeuk, Victor; Kanter, Julie; R. Abboud, Miguel; Lehrer‐Graiwer, Joshua; Tonda, Margaret; Intondi, Allison; Tong, Barbara; Howard, Jo

doi:10.1056/nejmoa1903212

Public

A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease

Published in New England Journal of Medicine • Jun 14, 2019

Authors:

Elliott Vichinsky

Carolyn Hoppe

Kenneth I. Ataga

Abstract

BACKGROUND: 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. METHODS: In a multicenter, phase 3, ...

Subject

Medicine

Placebo

Hemoglobin

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