eteplirsen
eteplirsen
A proprietary agent in clinical trials for managing Duchenne muscular dystrophy, a condition characterised by defective dystrophin—a key cytoplasmic protein that links muscle fibre cytoskeleton to extracellular matrix through the cell membrane.Mechanism
Eteplirsen is a splice-switching oligomer that skips exon 51 of the dystrophin gene-DMD, resulting in the production of a shorter, but still functional, dystrophin.