eteplirsen   Click here for help

GtoPdb Ligand ID: 7534

Synonyms: AVI-4658 | Exondys 51®
Approved drug
eteplirsen is an approved drug (FDA (2016))
Compound class: Synthetic organic
Comment: Eteplirsen is a novel oligonucleotide drug for the treatment of Duchenne muscular dystrophy (DMD), caused by specific reading frame mutations in the dystrophin gene [2]. Structurally it is a 30 base morpholino antisense oligomer in which a six-membered morpholine ring replaces the ribose sugars, and a phosphorodiamidate group replaces the normal anionic phosphodiester links between bases. This type of nucleotide is sometimes referred to as a phosphorodiamidate morpholino oligo, or PMO. While the size of this causes molecular indexing and rendering problems, two images of the chemical structure were found. The first from the NIH US National Library of Medicine where they provide the InChIKey KZOVCSBYYXUVDW-UHFFFAOYSA-N. The second is ChemSpider 34983391 with a different InChIKey GHBMWAGCLOEKQK-BMBJILFMSA-N.
2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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