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1. (WO2010116141) DRUG COMBINATION FOR THE TREATMENT OF PROTEOSTATIC DISEASES

Pub. No.:    WO/2010/116141    International Application No.:    PCT/GB2010/000710
Publication Date: Fri Oct 15 01:59:59 CEST 2010 International Filing Date: Thu Apr 08 01:59:59 CEST 2010
IPC: A61K 31/70
A61K 31/7016
A61K 31/702
A61P 3/00
A61P 25/00
Applicants: SUMMIT CORPORATION PLC
WILSON, Francis Xavier
STORER, Richard
KAWAMURA, Akane
NASH, Robert
ROACH, Alan Geoffrey
TINSLEY, Jonathon Mark
Inventors: WILSON, Francis Xavier
STORER, Richard
KAWAMURA, Akane
NASH, Robert
ROACH, Alan Geoffrey
TINSLEY, Jonathon Mark
Title: DRUG COMBINATION FOR THE TREATMENT OF PROTEOSTATIC DISEASES
Abstract:
Combinations comprising: (a) an active-site-specific pharmacoperone (ASSC) of an enzyme (e.g. a lysosomal enzyme); and (b) a non-active-site-specific pharmacoperone (non-ASSC) of said enzyme which: (i) does not bind to a catalytic site of said enzyme; or (ii) is not a competitive inhibitor of said enzyme; or (iii) is an activator of said enzyme; or (iv) is a non-competitive inhibitor of said enzyme; or (v) binds (e.g. specifically) to an allosteric site of said enzyme; or (vi) does not bind to said enzyme but binds to a chaperone or co- chaperone of said enzyme are described The combinations have various uses in medicine, including the treatment of protein folding diseases (in particular lysosomal storage disorders).