GOLODIRSEN

Approval Year

Substance Class	Nucleic Acid
Nucleic Acid Type	OLIGONUCLEOTIDE
Nucleic Acid Subtype	Other EXON-SKIPPING
Sequence Type	COMPLETE
Record UNII	033072U4MZ
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

GOLODIRSEN

Official Name

English

golodirsen [INN]

Common Name

English

NG-12-0163

Code

English

GOLODIRSEN [MI]

Common Name

English

Golodirsen [WHO-DD]

Common Name

English

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Classification Tree

Code System

Code

Designated/Approved

FDA ORPHAN DRUG

636818

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Code System

Code

Type

Description

DRUG BANK

DB15593

PRIMARY

WIKIPEDIA

Golodirsen

PRIMARY

FDA UNII

033072U4MZ

PRIMARY

CAS

1422959-91-8

PRIMARY

RXCUI

2267207

PRIMARY

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Subunit 0

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Sugar	Site Range	Site Count
morpholino	1_1-1_25	25 / 25

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Linkage	Site Range	Site Count
Phosphoryldimethylamine	1_2-1_25	24 / 24

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Related Record

Type

Details


					60RX8439K2

DYSTROPHIN

TARGET -> INDUCER

Comments:

exon 53 skipping

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Related Record

Type

Details


					033072U4MZ

GOLODIRSEN

ACTIVE MOIETY

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Structural Modifications

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Modification Type	Location Site	Location Type	Residue Modified	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

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Name	Property Type	Amount	Referenced Substance	Defining	Parameters	References
MOL_WEIGHT:NUMBER(CALCULATED)	CHEMICAL	NUMBER

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