Emerging Therapies in β-Thalassemia
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W.B. Saunders
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Keywords
Advancement of care, Clinical trial, Novel therapies, Quality of life, Transfusion burden, Β-thalassemia, Beta-thalassemia, Erythropoiesis, Humans, Iron, Iron overload, Artificial enzyme, Benserazide, Bitopertin, Enzyme activator, Erythroid maturation agent, Etavopivat, Ferroportin inhibitor, Glycine transporter 1 inhibitor, Hemoglobin alpha chain, Hemoglobin beta chain, Hemoglobin f, Hepcidin mimetics, Hydroxyurea, Janus kinase 2 inhibitor, Luspatercept, Mitapivat, Placebo, Protein inhibitor, Pyruvate kinase activator, Ruxolitinib, Sirolimus, Small interfering rna, Sotatercept, Thalidomide, Tmprss6 protein, Tovinontrine, Unclassified drug, Vamifeport, Anemia, Antiinflammatory activity, Beta thalassemia, Bone density, Combination chemotherapy, Dizziness, Dose response, Drug approval, Drug efficacy, Drug safety, Drug tolerability, Gene editing, Gene therapy, Hemolysis, Human, Insomnia, Monotherapy, Pharmacodynamics, Phase 1 clinical trial (topic), Phase 2 clinical trial (topic), Phase 3 clinical trial (topic), Practice guideline, Review, Tmprss6 gene, Treatment duration