筋ジストロフィーの治療法開発オーバービュー

抄録

Muscular dystrophy is a disease characterized by progressive muscle weakness. Patients require a multidisciplinary, long–term management, and currently there is no curative treatment.

Novel therapies have been rigorously investigated in recent years, and an exon skipping drug was approved in Japan for patients amenable to exon 53 skipping. Further therapeutic approaches including exon skipping, gene therapies, small molecules are also being studied, and the results of efficacy and safety are awaited.

Clinical trials in myotonic dystrophy, limb–girdle muscular dystrophy, facioscapulohumeral muscular dystrophy, and Fukuyama type congenital muscular dystrophy are also actively underway.

In this article, the current status of drug development for muscular dystrophy is reviewed.