METHOD OF TREATING GENETIC DISEASE CAUSED BY NONSENSE MUTATION

摘要

An object of the present invention is to provide compounds having read-through activity for use in treatment methods of genetic diseases caused by nonsense mutation, to provide pharmaceutical compositions comprising the compound, and to provide a treatment method of genetic diseases caused by nonsense mutation comprising administering the compound.;The present invention can provide a method of producing wild type normal protein in a living body of a mammal from a gene with a premature termination codon being generated by a mutation, wherein the method comprises administering a compound expressed by the following formula (VI):(wherein R¹, R², R³, R⁴, R⁵ and X¹ in the formula are as defined in description) or the like to the mammal.