Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules

Int J Mol Sci. 2019 Aug 17;20(16):4017. doi: 10.3390/ijms20164017.


This review, one in a series on myotonic dystrophy (DM), is focused on the development and potential use of small molecules as therapeutics for DM. The complex mechanisms and pathogenesis of DM are covered in the associated reviews. Here, we examine the various small molecule approaches taken to target the DNA, RNA, and proteins that contribute to disease onset and progression in myotonic dystrophy type 1 (DM1) and 2 (DM2).

Keywords: myotonic dystrophy; small molecules; therapies.

Publication types

  • Review

MeSH terms

  • Animals
  • Humans
  • Myotonic Dystrophy / drug therapy*
  • Myotonic Dystrophy / metabolism
  • Myotonic Dystrophy / therapy
  • RNA, Messenger / antagonists & inhibitors*


  • RNA, Messenger