Internal medicine

Disease

Bioinformatics

Biology

Drug repositioning

Authorization

Ecology

Myotonic dystrophy

Drug

Computer science

Repurposing

Drug development

Drug discovery

Computer security

Clinical trial

Medicine

Orphan drug

Pharmacology

Drug Discovery Today

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.

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