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http://dx.doi.org/10.22742/JIG.2021.3.2.25

New Drug Development of Myotonic Muscular Dystrophy  

Kang, Min Sung (Department of Neurology, Pusan National University Yangsan Hospital)
Shin Jin-Hong (Department of Neurology, Pusan National University Yangsan Hospital)
Publication Information
Journal of Interdisciplinary Genomics / v.3, no.2, 2021 , pp. 25-29 More about this Journal
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;
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