Chemical molecules that inhibit the slicing mechanism for treating diseases resulting from splicing anomalies
申请人:CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
公开号:US10654813B2
公开(公告)日:2020-05-19
Compounds and pharmaceutically acceptable salts thereof that may be used to treat a disease, for example, Duchenne muscular dystrophy, AIDS, and progeria. The compounds and pharmaceutically acceptable salts thereof may be part of a pharmaceutical composition including a pharmaceutically acceptable support.
可用于治疗疾病(如杜氏肌肉萎缩症、艾滋病和早衰症)的化合物及其药学上可接受的盐类。这些化合物及其药学上可接受的盐可以是包括药学上可接受的支持物的药物组合物的一部分。