Compounds for Nonsense Suppression, and Methods for Their Use
申请人:Almstead Neil G.
公开号:US20090253699A1
公开(公告)日:2009-10-08
The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA.
[EN] MODULATORS OF CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR<br/>[FR] MODULATEURS DU RÉGULATEUR DE LA CONDUCTANCE TRANSMEMBRANAIRE DE LA FIBROSE KYSTIQUE
申请人:VERTEX PHARMA
公开号:WO2022076629A1
公开(公告)日:2022-04-14
This disclosure provides modulators of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) having the structure: (I), pharmaceutical compositions, containing at least one such modulator, methods of treatment of cystic fibrosis using such modulators and pharmaceutical compositions, combination therapies, and processes and intermediates for making such modulators.
Pyrazole or triazole compounds and their use for the manufacture of a medicament for treating somatic mutation-related diseases
申请人:PTC Therapeutics, Inc.
公开号:EP2301536A1
公开(公告)日:2011-03-30
The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA.
COMPOUNDS FOR NONSENSE SUPPRESSION, AND METHODS FOR THEIR USE
申请人:PTC Therapeutics, Inc.
公开号:EP2939674A1
公开(公告)日:2015-11-04
The present invention relates to methods of Formula (1-A)
, and such compounds for use in the treatment or prevention of diseases associated with nonsense mutations in an mRNA, such as for suppressing premature translation termination associated with a nonsense mutation in an mRNA.