REGULATION OF CHOLESTEROL HOMEOSTASIS

摘要

The invention provides novel compounds of Formulas (I)-(IV), as described herein. Also provided are compositions of these compounds, method of making the compounds, and methods of using the compounds. The compounds can be used to regulate cholesterol homeostasis and to treat conditions and diseases associated with cholesterol homeostasis, including lysosomal lipid storage disorders such as Niemann-Pick Disease type C.

主权项

1. A compound of Formula (I): wherein
X is H, OH, or alkoxy;R1 is H, alkyl, cycloalkyl, amino, aryl, or heterocycle;R2 is H or CH3;R3 is H or CH3;R4 is H or CH3; provided that one of R3 and R4 is H; or a pharmaceutically acceptable salt thereof.