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COMPOUNDS FOR USES IN PHARMACOLOGICAL INDUCTION OF HBF FOR TREATMENT OF SICKLE CELL DISEASE AND β-THALASSEMIA
COMPOUNDS FOR USES IN PHARMACOLOGICAL INDUCTION OF HBF FOR TREATMENT OF SICKLE CELL DISEASE AND β-THALASSEMIA
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机译:药物用于HBF的药理学诱导治疗S细胞病和β地中海贫血
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摘要
Provided herein are compounds of Formula (I). The compounds described herein are useful in treating a disease associated with the expression of endogenous embryonic/fetal globin (e.g., γ globin) in erythrocytes (e.g., treating β -thalassemia and/or sickle cell disease through at least the induction of the globin gene expression in erythrocytes), and/or anemia (β-thalassemia and/or sickle cell anemia). The compounds described herein are useful in treating, delaying, and/or preventing the adverse effects of β-thalassemia and/or sickle cell disease, inducing γ globin production, and/or inducing the expression of embryonic/fetal globin genes in a subject, cell, tissue, or biological sample. Also provided in the present disclosure are pharmaceutical compositions, kits, and methods of using the compounds for inducing γ globin production described herein and for treating any of the target diseases described herein.
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