Ligand id: 7534

Name: eteplirsen

Structure and Physico-chemical Properties

2D Structure
No information available.
Summary of Clinical Use
In September 2016, the US FDA approved eteplirsen for DMD patients carrying mutations amenable to exon 51 skipping.
Mechanism Of Action and Pharmacodynamic Effects
The lack of dystrophin protein which gives rise to the fatal X-linked disease, Duchenne muscular dystrophy (DMD) is caused by mutations that disrupt the gene's reading frame and prevent full protein translation. Eteplirsen is designed to correct a specific exon 51 mutation, by annealing to a splicing element in the dystrophin mRNA which promotes skipping of exon 51 [1]. The aim is to restore the open reading frame for improved protein synthesis [2].