PATNAIK, Samarjit;THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES;SIDRANSKY, Ellen; AS REPRESENTED BY THE SECRETARY;MARUGAN, Juan Jose;SOUTHALL, Noel;THE UNITED STATES OF AMERICA;ZHENG, Wei;MOTA;GOLDIN, Ehud; DEPARTMENT OF HEALTH AND HUMAN SERVICES
发明人:
MARUGAN, Juan Jose,SOUTHALL, Noel,GOLDIN, Ehud,PATNAIK, Samarjit,SIDRANSKY, Ellen,MOTABAR, Omid,WESTBROOK, Wendy
申请号:
USUS2011/063928
公开号:
WO2012/078855A1
申请日:
2011.12.08
申请国别(地区):
US
年份:
2012
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 R1-R7 and X are described in the application.Cette invention concerne des pyrazolopyrimidines substituées et des dihydropyrazolopyrimidines et composés associés, leurs procédés de fabrication, les compositions contenant ces composés, et les procédés d'utilisation de ces composés pour traiter les troubles de stockage dans les lysosomes, notamment la maladie de Gaucher. Les composés sont de formule générale (I), les variables R1-R7 et X étant définies dans la description.