eteplirsen

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eteplirsen2016-12-03T13:15:11+00:00

eteplirsen 

PAEDIATRIC NEUROLOGY

A proprietary (Sarepta Therapeutics) agent in clinical trials for managing Duchenne muscular dystrophy, which is characterised by defective dystrophin–a 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 truncated–but still functional–dystrophin

Synonym AVI-4658 

References www.duchenneconnect.org

www.avibio.com 

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