A pharmaceutical composition for genetic diseases caused by an aberrant splicing regulation is provided.Provided are a pharmaceutical composition for preventing, ameliorating, suppressing progression of, and/or treating the genetic diseases caused by an aberrant splicing regulation, the pharmaceutical composition containing, as an active ingredient, a compound capable of suppressing an aberrant splicing regulation that contributes to the development or progression of genetic diseases caused by an aberrant splicing regulation, and a method for preventing, ameliorating, suppressing progression of, and/or treating the genetic diseases using a compound capable of suppressing an aberrant splicing regulation that contributes to the development or progression of genetic diseases caused by an aberrant splicing regulation.
