Journal of Interdisciplinary Genomics

Interdisciplinary Society of Genetic & Genomic Medicine (유전의학융합회)
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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)
  • Received : 2021.07.06
  • Accepted : 2021.07.27
  • Published : 2021.10.31
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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.

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References

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