KEGG   DRUG: EteplirsenHelp
Entry
D09900                      Drug                                   

Name
Eteplirsen (USAN/INN);
Exondys 51 (TN)
Product
Formula
C364H569N177O122P30
Exact mass
10300.589
Mol weight
10305.7382
Sequence
cuccaacauc aaggaagaug gcauuucuag
Remark
ATC code: M09AX06
Product: D09900<US>
Efficacy
Translation inhibitor
  Disease
Duchenne muscular dystrophy (exon 51 skipping) [DS:H01963]
Comment
Phosphorodiamidate morpholino oligomer (PMO): synthetic DNA analogs
Antisense oligonucleotide; DMD pre-mRNA (exon51) [HSA:1756]
Eteplirsen induces skipping of dystrophin [HSA:1756] [KO:K10366] exon 51.
Treatment of Duchenne muscular dystrophy
Target
DMD [HSA_VAR:1756v1] [HSA:1756] [KO:K10366]
Brite
Anatomical Therapeutic Chemical (ATC) classification [BR:br08303]
 M MUSCULO-SKELETAL SYSTEM
  M09 OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
   M09A OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
    M09AX Other drugs for disorders of the musculo-skeletal system
     M09AX06 Eteplirsen
      D09900  Eteplirsen (USAN/INN) <US>
USP drug classification [BR:br08302]
 Genetic or Enzyme Disorder: Replacement, Modifiers, Treatment
  Eteplirsen
   D09900  Eteplirsen (USAN/INN)
Target-based classification of drugs [BR:br08310]
 Not elsewhere classified
  Cellular components
   Cytoskeleton
    DMD [HSA_VAR:1756v1]
     D09900  Eteplirsen (USAN/INN) <US>
New drug approvals in the USA [br08319.html]
 New Molecular Entity and New Therapeutic Biological Product Approvals
  D09900
New drug approvals in the USA, Europe and Japan [br08328.html]
 Approval dates by FDA, EMA and PMDA
  D09900
Pharmacogenomic biomarkers [br08341.html]
 Companion diagnostics
  D09900
BRITE hierarchy
Other DBs
CAS: 1173755-55-9
PubChem: 124490612
LinkDB All DBs

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