compound of the formula, pharmaceutical composition, method of treating gaucher disease and method of increasing the amount of beta glucocerebrosidase in white blood cells.
THE UNITED STATES OF AMERICA; AS REPRESENTED BY THE SECRETARY; DEPARTMENT OF HEALTH AND HUMAN SERVICES
发明人:
EHUD GOLDIN,ELLEN SIDRANSKY,JUAN JOSE MARUGAN,NOEL SOUTHALL,OMID MOTABAR,SAMARJIT PATNAIK,WEI ZHENG,WENDY WESTBROOK
申请号：
BR112013014242
公开号：
BR112013014242A2
申请日:
2011.12.08
申请国别(地区):
BR
年份:
2018
代理人:
摘要：
Substituted pyrazolopyrimidines and dihydropyrazolopyrimidines and related compounds, their methods of manufacture, compositions containing these compounds, and methods of use of these compounds in treating lysosomal storage disorders such as Gaucher disease are described herein. The compounds are of general Formula (I) in which variables R 1 -R 7 and X are described in the application.
