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Synonyms: AVI-4658 | Exondys 51®
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 . 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 and they provide the InChIKey KZOVCSBYYXUVDW-UHFFFAOYSA-N. The second is ChemSpider 34983391 with a different InChIKey GHBMWAGCLOEKQK-BMBJILFMSA-N
1. Anthony K, Feng L, Arechavala-Gomeza V, Guglieri M, Straub V, Bushby K, Cirak S, Morgan J, Muntoni F. (2012)
Exon skipping quantification by quantitative reverse-transcription polymerase chain reaction in Duchenne muscular dystrophy patients treated with the antisense oligomer eteplirsen.
Hum Gene Ther Methods, 23 (5): 336-45. [PMID:23075107]
2. Kinali M, Arechavala-Gomeza V, Feng L, Cirak S, Hunt D, Adkin C, Guglieri M, Ashton E, Abbs S, Nihoyannopoulos P et al.. (2009)
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study.
Lancet Neurol, 8 (10): 918-28. [PMID:19713152]