MONARSEN

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Tue Apr 01 20:12:12 GMT 2025` Edited by `admin` on `Tue Apr 01 20:12:12 GMT 2025`
Nucleic Acid Type	OLIGONUCLEOTIDE
Sequence Origin	HUMAN
Sequence Type	COMPLETE
Record UNII	4TT3V55XYT
Record Status	`Validated (UNII)`
Record Version

Download

Name

Type

Language

EN-101

Preferred Name

English

MONARSEN

Common Name

English

BL-7040

Code

English

0DN-7040

Common Name

English

DNA, D(C-T-G-C-G-A-T-A-T-T-T-T-C-T-T-G-T-RAM-RCM-RCM)

Common Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

173903

Created by admin on Tue Apr 01 20:12:12 GMT 2025 , Edited by admin on Tue Apr 01 20:12:12 GMT 2025

Code System Code Type Description

CAS

864021-55-6

Created by admin on Tue Apr 01 20:12:12 GMT 2025 , Edited by admin on Tue Apr 01 20:12:12 GMT 2025

PRIMARY

FDA UNII

4TT3V55XYT

Created by admin on Tue Apr 01 20:12:12 GMT 2025 , Edited by admin on Tue Apr 01 20:12:12 GMT 2025

PRIMARY

SMS_ID

300000022451

Created by admin on Tue Apr 01 20:12:12 GMT 2025 , Edited by admin on Tue Apr 01 20:12:12 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
2'-O-Methyl-Ribose	1_18-1_20	3 / 20
Deoxyribose (DNA)	1_1-1_17	17 / 20

Linkage	Site Range	Site Count
Phosphate	1_2-1_20	19 / 19

Related Record Type Details


					EK71H4HC3N

ACETYLCHOLINESTERASE HUMAN

TARGET->INHIBITOR OF EXPRESSION

Amount:


					23MZ977QUX

MONARSEN SODIUM

SALT/SOLVATE -> PARENT

Amount:

Related Record Type Details


					4TT3V55XYT

MONARSEN

ACTIVE MOIETY

Comments:

Monarsen was shown to improve the Quantitativ emyasthenia gravis (QMG) score in 14 of the 16 patients examined,

Amount:

Name	Property Type	Amount	Referenced Substance	Defining	Parameters	References
MOL_WEIGHT	CHEMICAL