Translational medicine in neuromuscular disorders: from academia to industry

Dis Model Mech. 2019 Oct 24;13(2):dmm041434. doi: 10.1242/dmm.041434.


Although around half of US Food and Drug Administration (FDA)-approved drugs originate from discoveries made in academic research laboratories, the US National Institutes of Health (NIH) estimates that nearly 90% of therapies developed in preclinical stages never reach clinical trials. From those in clinical trials, only 10% obtain marketing approval. Despite the recent advances in our understanding and diagnosis of neuromuscular disease, and the development of rational therapies in clinical trials, these numbers have not changed dramatically over the past two decades. This article discusses the advantages and challenges for translational research initiated from academia, and the trend towards bridging the gap between discovery and translation to the clinic. A focus is made on recent advances in therapeutic development for neuromuscular disorders.

Keywords: Drug development; Neuromuscular disease; Therapy; Translational research.

Publication types

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

MeSH terms

  • Clinical Trials as Topic
  • Drug Development
  • Drug Industry*
  • Humans
  • Neuromuscular Diseases / drug therapy*
  • Translational Medical Research*
  • Universities*