METHODS AND COMPOSITIONS FOR TREATING beta-THALASSEMIA AND SICKLE CELL DISEASE
摘要
<p>The present invention provides compounds (such as compounds of formula I-a) that induce gamma 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 beta-thalassemia and sickle cell anemia. (I-a)</p>
