Teaching an Old Molecule New Tricks: Drug Repositioning for Duchenne Muscular Dystrophy.

ABSTRACT: : Duchenne muscular dystrophy (DMD) is one of the most severe forms of inherited muscular dystrophies. The disease is caused by the lack of dystrophin, a structurally essential protein; hence, a definitive cure would necessarily have to pass through some form of gene and/or cell therapy. Cell- and genetic-based therapeutics for DMD have been explored since the 1990s and recently, two of the latter have been approved for clinical use, but their efficacy is still very low. In parallel, there have been great ongoing efforts aimed at targeting the downstream pathogenic effects of dystrophin deficiency using classical pharmacological approaches, with synthetic or biological molecules. However, as it is always the case with rare diseases, R&D costs for new drugs can represent a major hurdle for

SUBMITTER: Vitiello L 

PROVIDER: S-EPMC6929176 | biostudies-literature | 2019 Nov

REPOSITORIES: biostudies-literature