monrd0013
Medals oligonucleic drug ID monrd0013
ATC Classification -
Product Name Eteplirsen
Synonims Exondys 51
Country US
Company Sarepta Therapeutics, Inc.
phase AD
Type Antisense
Adaptation Diseases Duchenne muscular dystrophy
Target Molecule DMD-001
Remarks Eteplirsen is a phosphoramidite morpholino sequence complementary to a portion of exon 51. It exerts it's mechanism of action by forcing the exclusion of exon 51 from the mature DMD mRNA.
Nucleic Acid Sequence 5'-CTCCAACATCAAGGAAGATGGCATTTCTAG-3'
Source https://www.sarepta.com/our-product,
https://www.drugbank.ca/drugs/DB06014,
http://www.rxlist.com/exondys-51-drug.htm,
https://www.drugs.com/ingredient/eteplirsen.html
DrugBank DB06014
TargetProtein - UniProt P11532
TargetProtein - PDB 1EG3