COMPOUND TREATING AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEMENTIA AND APPLICATION THEREOF

摘要

The present invention relates to a compound for treating amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) and an application of the compound. The invention discloses a compound suppressing a toxicity induced by an rGGGGCC repeat segment in a first intron of C9ORF72 and an application of the compound in preparing a pharmaceutical drug for treating ALS and FTD. The compound suppressing the toxicity induced by the rGGGGCC repeat segment in the first intron of C9ORF72 can significantly improve dyskinesias induced by the rGGGGCC repeat segment.