Abstract
Preclinical data demonstrating that luspatercept mitigates vaso-occlusive and anemia-related complications in the Townes sickle cell model provide a strong rationale for its evaluation in SCD patients.
Keywords:
Hematology; Hematopoietic stem cells; Immunology.
MeSH terms
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Activin Receptors, Type II
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Anemia, Sickle Cell* / complications
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Anemia, Sickle Cell* / drug therapy
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Anemia, Sickle Cell* / genetics
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Anemia, Sickle Cell* / pathology
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Animals
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Disease Models, Animal
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Humans
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Immunoglobulin Fc Fragments* / pharmacology
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Mice
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Phenotype
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Recombinant Fusion Proteins* / pharmacology
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Vaso-Occlusive Crises
Substances
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Recombinant Fusion Proteins
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luspatercept
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Immunoglobulin Fc Fragments
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Activin Receptors, Type II