Myotonic dystrophy type 1 drug development: A pipeline toward the market

Drug Discov Today. 2021 Jul;26(7):1765-1772. doi: 10.1016/j.drudis.2021.03.024. Epub 2021 Mar 31.


Myotonic dystrophy type 1 (DM1) is a multisystemic neuromuscular genetic disease with an estimated prevalence of approximately at least half a million individuals based on its vast ethnic variation. Building upon a well-known physiopathology and several proof-of-concept therapeutic approaches, herein we compile a comprehensive overview of the most recent drug development programs under preclinical and clinical evaluation. Specifically, close to two dozen drug developments, eight of which are already in clinical trials, explore a diversity of new chemical entities, drug repurposing, oligonucleotide, and gene therapy-based approaches. Of these, repurposing of tideglusib, mexiletine, or metformin appear to be therapies with the most potential to receive marketing authorization for DM1.

Keywords: Antisense oligonucleotide; Clinical trial; Drug development; Gene therapy; Myotonic dystrophy; Repurposing drug.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Drug Development
  • Humans
  • Myotonic Dystrophy / drug therapy*