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Substance Name: Golodirsen [USAN:INN]
RN: 1422959-91-8
UNII: 033072U4MZ


  • Synthetic antisense oligonucleotide designed to cause skipping of abnormal exons in the synthesis of the dystrophin gene and that is used to treat Duchenne muscular dystrophy.

Molecular Formula

  • C305-H481-N138-O112-P25

Classification Codes

  • Molecular Probes
  • Oligonucleotides, Antisense
  • Treatment of Duchenne Muscular Dystrophy
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Names and Synonyms

Results Name

  • Golodirsen [USAN:INN]

Name of Substance

  • Golodirsen
  • Golodirsen [USAN]


  • Golodirsen
  • Golodirsen [INN]
  • NG-12-0163
  • Rna, (p-deoxy-p-(dimethylamino))(2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a->5')(g-m5u-m5u-g-c-c-m5u-c-c-g-g-m5u-m5u-c-m5u-g-a-a-g-g-m5u-g-m5u-m5u-C), 5'-(p-(4-((2-(2-(2-hydroxyethoxy)ethoxy)ethoxy)carbonyl)-1-piperazinyl)-N,N-dimethylphosphonamidate)
  • SRP-4053
  • UNII-033072U4MZ
  • WHO 10355

Registry Numbers

CAS Registry Number

  • 1422959-91-8


  • 033072U4MZ

System Generated Number

  • 1422959918