GOLODIRSEN

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 `Wed Apr 02 08:42:36 GMT 2025` Edited by `admin` on `Wed Apr 02 08:42:36 GMT 2025`
Nucleic Acid Type	OLIGONUCLEOTIDE
Sequence Type	COMPLETE
Record UNII	033072U4MZ
Record Status	`Validated (UNII)`
Record Version

Download

Name

Type

Language

NG-12-0163

Preferred Name

English

GOLODIRSEN

Official Name

English

golodirsen [INN]

Common Name

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 Wed Apr 02 08:42:36 GMT 2025 , Edited by admin on Wed Apr 02 08:42:36 GMT 2025

Code System Code Type Description

DRUG BANK

DB15593

Created by admin on Wed Apr 02 08:42:36 GMT 2025 , Edited by admin on Wed Apr 02 08:42:36 GMT 2025

PRIMARY

WIKIPEDIA

Golodirsen

Created by admin on Wed Apr 02 08:42:36 GMT 2025 , Edited by admin on Wed Apr 02 08:42:36 GMT 2025

PRIMARY

FDA UNII

033072U4MZ

Created by admin on Wed Apr 02 08:42:36 GMT 2025 , Edited by admin on Wed Apr 02 08:42:36 GMT 2025

PRIMARY

CAS

1422959-91-8

Created by admin on Wed Apr 02 08:42:36 GMT 2025 , Edited by admin on Wed Apr 02 08:42:36 GMT 2025

PRIMARY

RXCUI

2267207

Created by admin on Wed Apr 02 08:42:36 GMT 2025 , Edited by admin on Wed Apr 02 08:42:36 GMT 2025

PRIMARY

DAILYMED

033072U4MZ

Created by admin on Wed Apr 02 08:42:36 GMT 2025 , Edited by admin on Wed Apr 02 08:42:36 GMT 2025

PRIMARY

MERCK INDEX

m12185

Created by admin on Wed Apr 02 08:42:36 GMT 2025 , Edited by admin on Wed Apr 02 08:42:36 GMT 2025

PRIMARY

DRUG CENTRAL

5357

Created by admin on Wed Apr 02 08:42:36 GMT 2025 , Edited by admin on Wed Apr 02 08:42:36 GMT 2025

PRIMARY

USAN

FG-219

Created by admin on Wed Apr 02 08:42:36 GMT 2025 , Edited by admin on Wed Apr 02 08:42:36 GMT 2025

PRIMARY

SMS_ID

100000174615

Created by admin on Wed Apr 02 08:42:36 GMT 2025 , Edited by admin on Wed Apr 02 08:42:36 GMT 2025

PRIMARY

EPA CompTox

DTXSID401027895

Created by admin on Wed Apr 02 08:42:36 GMT 2025 , Edited by admin on Wed Apr 02 08:42:36 GMT 2025

PRIMARY

INN

10355

Created by admin on Wed Apr 02 08:42:36 GMT 2025 , Edited by admin on Wed Apr 02 08:42:36 GMT 2025

PRIMARY

NCI_THESAURUS

C175077

Created by admin on Wed Apr 02 08:42:36 GMT 2025 , Edited by admin on Wed Apr 02 08:42:36 GMT 2025

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


					60RX8439K2

TARGET -> INDUCER

Comments:

exon 53 skipping

Related Record Type Details


					033072U4MZ

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