Methods for treating nonsense mutation hereditary diseases

摘要

An object of the present invention is to provide a compound having read-through activity for use as a method for treating a nonsense mutant genetic disease, to provide a pharmaceutical composition containing the compound, and to administer the compound A method of treating a nonsense mutant genetic disease comprising: According to the present invention, there is provided a method for producing a wild-type normal protein in vivo in a mammal from a gene in which an immature stop codon is generated by mutation, wherein the following formula (VI): [wherein R1, R2, R3, R4, R5 And X1 is as defined in the specification], and the like.