New drug development of myotonic muscular dystrophy

Min Sung Kang (Department of Neurology, Pusan National University Yangsan Hospital, Yangsan, Korea)
Jin-Hong Shin (Department of Neurology, Pusan National University Yangsan Hospital, Yangsan, Korea)

Kang, M. S., & Shin, J. (2021). . 유전의학융합학술지, 3(2), 25-29, https://doi.org/10.22742/JIG.2021.3.2.25

복사

Abstract

Myotonic muscular dystrophy is a disease characterized by progressive muscle weakness with myotonia and multiorgan involvement. Two subtypes have been recognized; each subtype is caused by nucleotide repeat expansion. So far, there has been no cure for myotonic muscular dystrophy. In this article, we introduce ongoing clinical trials for new drugs to modify disease course by correcting genetic derangement or its downstream in myotonic dystrophy type 1.

keywords: Myotonic Dystrophy, Genetic Therapy, Drug Development, Muscular Dystrophies, Oligonucleotides

투고일Submission Date: 2021-07-06

수정일Revised Date: 2021-07-24

게재확정일Accepted Date: 2021-07-27

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논문 상세

Vol.3 No.2

New drug development of myotonic muscular dystrophy

Abstract

유전의학융합학술지