U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Approval Year

Substance Class Nucleic Acid
Created
by admin
on Thu Jul 06 22:27:45 UTC 2023
Edited
by admin
on Thu Jul 06 22:27:45 UTC 2023
Nucleic Acid Type OLIGONUCLEOTIDE
Nucleic Acid Subtype Other EXON-SKIPPING
Sequence Type COMPLETE
Record UNII
033072U4MZ
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
GOLODIRSEN
INN  
INN  
Official Name English
golodirsen [INN]
Common Name English
NG-12-0163
Code English
GOLODIRSEN [MI]
Common Name English
Golodirsen [WHO-DD]
Common Name English
GOLODIRSEN [USAN]
Common Name English
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)
Systematic Name English
GOLODIRSEN [ORANGE BOOK]
Common Name English
VYONDYS 53
Brand Name English
SRP-4053
Code English
Classification Tree Code System Code
FDA ORPHAN DRUG 636818
Created by admin on Thu Jul 06 22:27:45 UTC 2023 , Edited by admin on Thu Jul 06 22:27:45 UTC 2023
Code System Code Type Description
DRUG BANK
DB15593
Created by admin on Thu Jul 06 22:27:45 UTC 2023 , Edited by admin on Thu Jul 06 22:27:45 UTC 2023
PRIMARY
WIKIPEDIA
Golodirsen
Created by admin on Thu Jul 06 22:27:45 UTC 2023 , Edited by admin on Thu Jul 06 22:27:45 UTC 2023
PRIMARY
FDA UNII
033072U4MZ
Created by admin on Thu Jul 06 22:27:45 UTC 2023 , Edited by admin on Thu Jul 06 22:27:45 UTC 2023
PRIMARY
CAS
1422959-91-8
Created by admin on Thu Jul 06 22:27:45 UTC 2023 , Edited by admin on Thu Jul 06 22:27:45 UTC 2023
PRIMARY
RXCUI
2267207
Created by admin on Thu Jul 06 22:27:45 UTC 2023 , Edited by admin on Thu Jul 06 22:27:45 UTC 2023
PRIMARY
DAILYMED
033072U4MZ
Created by admin on Thu Jul 06 22:27:45 UTC 2023 , Edited by admin on Thu Jul 06 22:27:45 UTC 2023
PRIMARY
MERCK INDEX
M12185
Created by admin on Thu Jul 06 22:27:45 UTC 2023 , Edited by admin on Thu Jul 06 22:27:45 UTC 2023
PRIMARY
DRUG CENTRAL
5357
Created by admin on Thu Jul 06 22:27:45 UTC 2023 , Edited by admin on Thu Jul 06 22:27:45 UTC 2023
PRIMARY
USAN
FG-219
Created by admin on Thu Jul 06 22:27:45 UTC 2023 , Edited by admin on Thu Jul 06 22:27:45 UTC 2023
PRIMARY
EPA CompTox
DTXSID401027895
Created by admin on Thu Jul 06 22:27:45 UTC 2023 , Edited by admin on Thu Jul 06 22:27:45 UTC 2023
PRIMARY
INN
10355
Created by admin on Thu Jul 06 22:27:45 UTC 2023 , Edited by admin on Thu Jul 06 22:27:45 UTC 2023
PRIMARY
NCI_THESAURUS
C175077
Created by admin on Thu Jul 06 22:27:45 UTC 2023 , Edited by admin on Thu Jul 06 22:27:45 UTC 2023
PRIMARY
Sugar Site Range Site Count
morpholino 1_1-1_25 25 / 25
Linkage Site Range Site Count
Phosphoryldimethylamine 1_2-1_25 24 / 24
Related Record Type Details
TARGET -> INDUCER
exon 53 skipping
Related Record Type Details
ACTIVE MOIETY

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
NUCLEOSIDE_SUBSTITUTION [1_17] [1_18] SITE_SPECIFIC A MORPHOLINO ADENOSINE 3ZYB3DY5HB
NUCLEOSIDE_SUBSTITUTION [1_5] [1_6] [1_8] [1_9] [1_14] [1_25] SITE_SPECIFIC MORPHOLINO CYTIDINE KFE1UKU3TI
NUCLEOTIDE SUBSTITUTION [1_1] 5'-TERMINUS GOLODIRSEN 5'-GUANINE MODIFICATION K9SST61XV5
NUCLEOSIDE_SUBSTITUTION [1_4] [1_10] [1_11] [1_16] [1_19] [1_20] [1_22] SITE_SPECIFIC MORPHOLINO GUANOSINE XHA1WD2RB2
NUCLEOSIDE_SUBSTITUTION [1_2] [1_3] [1_7] [1_12] [1_13] [1_15] [1_21] [1_23] [1_24] SITE_SPECIFIC MORPHOLINO THYMIDINE YML1Y6H5HD
Name Property Type Amount Referenced Substance Defining Parameters References
MOL_WEIGHT:NUMBER(CALCULATED) CHEMICAL