筋強直性ジストロフィーの治療開発と将来展望

抄録

Myotonic dystrophy type 1 (DM1) is an autosomal dominant neuromuscular disorder and ranks among the most prevalent muscle diseases. The root cause of DM1 is identified as RNA toxicity induced by abnormal RNAs stemming from the genetic mutation. Despite the absence of a cure for DM1, there is ongoing progress in the development of therapies targeting these deviant RNAs, which are approaching the final stages of commercialization.