Emerging Therapies in β-Thalassemia

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dc.contributor.authorBou-Fakhredin, Rayan
dc.contributor.authorKuo, Kevin H.M.
dc.contributor.authorTaher, Ali T.
dc.contributor.departmentInternal Medicine
dc.contributor.departmentDivision of Hematology Oncology
dc.contributor.facultyFaculty of Medicine (FM)
dc.contributor.institutionAmerican University of Beirut
dc.date.accessioned2025-01-24T11:45:01Z
dc.date.available2025-01-24T11:45:01Z
dc.date.issued2023
dc.identifier.doihttps://doi.org/10.1016/j.hoc.2022.12.010
dc.identifier.eid2-s2.0-85150225825
dc.identifier.pmid36907614
dc.identifier.urihttp://hdl.handle.net/10938/30520
dc.language.isoen
dc.publisherW.B. Saunders
dc.relation.ispartofHematology/Oncology Clinics of North America
dc.sourceScopus
dc.subjectAdvancement of care
dc.subjectClinical trial
dc.subjectNovel therapies
dc.subjectQuality of life
dc.subjectTransfusion burden
dc.subjectΒ-thalassemia
dc.subjectBeta-thalassemia
dc.subjectErythropoiesis
dc.subjectHumans
dc.subjectIron
dc.subjectIron overload
dc.subjectArtificial enzyme
dc.subjectBenserazide
dc.subjectBitopertin
dc.subjectEnzyme activator
dc.subjectErythroid maturation agent
dc.subjectEtavopivat
dc.subjectFerroportin inhibitor
dc.subjectGlycine transporter 1 inhibitor
dc.subjectHemoglobin alpha chain
dc.subjectHemoglobin beta chain
dc.subjectHemoglobin f
dc.subjectHepcidin mimetics
dc.subjectHydroxyurea
dc.subjectJanus kinase 2 inhibitor
dc.subjectLuspatercept
dc.subjectMitapivat
dc.subjectPlacebo
dc.subjectProtein inhibitor
dc.subjectPyruvate kinase activator
dc.subjectRuxolitinib
dc.subjectSirolimus
dc.subjectSmall interfering rna
dc.subjectSotatercept
dc.subjectThalidomide
dc.subjectTmprss6 protein
dc.subjectTovinontrine
dc.subjectUnclassified drug
dc.subjectVamifeport
dc.subjectAnemia
dc.subjectAntiinflammatory activity
dc.subjectBeta thalassemia
dc.subjectBone density
dc.subjectCombination chemotherapy
dc.subjectDizziness
dc.subjectDose response
dc.subjectDrug approval
dc.subjectDrug efficacy
dc.subjectDrug safety
dc.subjectDrug tolerability
dc.subjectGene editing
dc.subjectGene therapy
dc.subjectHemolysis
dc.subjectHuman
dc.subjectInsomnia
dc.subjectMonotherapy
dc.subjectPharmacodynamics
dc.subjectPhase 1 clinical trial (topic)
dc.subjectPhase 2 clinical trial (topic)
dc.subjectPhase 3 clinical trial (topic)
dc.subjectPractice guideline
dc.subjectReview
dc.subjectTmprss6 gene
dc.subjectTreatment duration
dc.titleEmerging Therapies in β-Thalassemia
dc.typeReview

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