Invention Grant
US08822491B2 Methods and compositions for treating β-thalassemia and sickle cell disease 有权
用于治疗地中海贫血和镰状细胞病的方法和组合物

Methods and compositions for treating β-thalassemia and sickle cell disease
Abstract:
The present invention provides compounds (such as compounds of formula I-a) that induce γ globin expression and pharmaceutical compositions thereof. Such compounds may have beneficial therapeutic effects. Compounds and compositions described herein may be used to treat hemoglobinopathies such as β-thalassemia and sickle cell anemia.
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