Provided is a pharmaceutical composition for a hereditary disease caused by a splicing abnormality. Pharmaceutical composition for preventing, ameliorating, suppressing progression, and / or treating a hereditary disease, comprising as an active ingredient a compound capable of suppressing the splicing abnormality that contributes to the onset or progression of the hereditary disease caused by the splicing abnormality. A method for preventing, ameliorating, suppressing progression, and / or treating a hereditary disease using a substance and a compound capable of suppressing the splicing abnormality that contributes to the onset or progression of the hereditary disease caused by the splicing abnormality.スプライシング異常に起因する遺伝性疾病のための医薬組成物の提供。スプライシング異常に起因する遺伝性疾病の発症又は進行の一因となるスプライシング異常を抑制できる化合物を有効成分として含む、該遺伝性疾病の予防、改善、進行抑制、及び／又は治療のための医薬組成物、及びスプライシング異常に起因する遺伝性疾病の発症又は進行の一因となるスプライシング異常を抑制できる化合物を用いて、該遺伝性疾病を予防、改善、進行抑制及び／又は治療する方法。
