Patent ID: 9649314
Date: 2017-05-16
CPC Classifications: A61K,A61P,C07D

Claim:
1. A method of suppressing the effect of nonsense mutation in a subject with a disease caused by the nonsense mutation wherein the disease is selected from hemophilia A, hemophilia B, cystic fibrosis, or mucopolysaccharidosis I, comprising administering to the subject a therapeutically effective amount of a compound of formula (I) in free form or in pharmaceutically acceptable salt form which is wherein R and R or R or R or R wherein the phenyl ring is attached via the bond marked with an asterisk; each R and R or R or R R R and R or a three- to seven-membered monocyclic aromatic, saturated or unsaturated non-aromatic ring system, wherein said ring system may contain from 1 to 4 hetero atoms selected from nitrogen, oxygen and sulfur, wherein said ring system may be attached directly or via a C or R R R or C or two R or two R R or C or two R or two R