The Current State of Sickle Cell Disease Treatments [Part I]

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American Society of Hematology. Abstract. 2022. Available from: Paper:Lovo-cel(bb1111)GeneTherapyforSickleCellDisease:UpdatedClinicalResultsandInvestigationsintoTwoCasesofAnemiafromGroupCofthePhase1/2HGB-206Study(confex.com) [Last accessed: November 21, 2022].

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Gentinetta T , Belcher JD , Brügger-Verdon V , et al. Plasma-derived hemopexin as a candidate therapeutic agent for acute vaso-occlusion in sickle cell disease: Preclinical evidence. J Clin Med, 2022; 11(3):630; doi: 10.3390/jcm11030630

Forsyth D , Schroeder P , Geib J . Safety, pharmacokinetics, and pharmacodynamics of Etavopivat (FT-4202), an allosteric activator of pyruvate kinase-R, in healthy adults: A randomized, placebo-controlled, double-blind, first in-human phase 1 trial. Clin Pharmacol Drug Dev, 2022; 11(5) 654–665; doi: 10.1002/cpdd.1058

van Dijk MJ , Rab MAE , van Oirschot BA , et al. Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in sickle cell disease: A phase 2, open-label study. Am J Hematol, 2022; 97(7):E226–E229; doi: 10.1002/ajh.26554