Approval Year
| Substance Class |
Nucleic Acid
Created
by
admin
on
Edited
Tue Apr 01 21:26:42 GMT 2025
by
admin
on
Tue Apr 01 21:26:42 GMT 2025
|
| Nucleic Acid Type | OLIGONUCLEOTIDE |
| Sequence Type | COMPLETE |
| Record UNII |
AIW6036FAS
|
| Record Status |
Validated (UNII)
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| Record Version |
|
-
Download
| Name | Type | Language | ||
|---|---|---|---|---|
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Preferred Name | English | ||
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Official Name | English | ||
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Common Name | English | ||
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Common Name | English | ||
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Common Name | English | ||
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Code | English | ||
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Common Name | English | ||
|
Common Name | English |
| Classification Tree | Code System | Code | ||
|---|---|---|---|---|
|
WHO-ATC |
M09AX06
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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|
NDF-RT |
N0000191626
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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|
FDA ORPHAN DRUG |
248407
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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| Code System | Code | Type | Description | ||
|---|---|---|---|---|---|
|
CHEMBL2108278
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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PRIMARY | |||
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Exondys (Refused)
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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PRIMARY | |||
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100000155279
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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PRIMARY | |||
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5182
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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PRIMARY | |||
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C171739
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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PRIMARY | |||
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AIW6036FAS
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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PRIMARY | |||
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SUB129287
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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PRIMARY | |||
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9339
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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PRIMARY | |||
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1810569
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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PRIMARY | |||
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N0000011392
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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PRIMARY | Oligonucleotides, Antisense [Chemical/Ingredient] | ||
|
ETEPLIRSEN
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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PRIMARY | |||
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XX-107
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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PRIMARY | |||
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m12193
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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PRIMARY | |||
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DB06014
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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PRIMARY | |||
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AIW6036FAS
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
|
PRIMARY | |||
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DTXSID701027882
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
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PRIMARY | |||
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1173755-55-9
Created by
admin on Tue Apr 01 21:26:42 GMT 2025 , Edited by admin on Tue Apr 01 21:26:42 GMT 2025
|
PRIMARY |
| Sugar | Site Range | Site Count |
|---|---|---|
| morpholino | 1_1-1_30 | 30 / 30 |
| Linkage | Site Range | Site Count |
|---|---|---|
| Phosphoryldimethylamine | 1_2-1_30 | 29 / 29 |
| Related Record | Type | Details | ||
|---|---|---|---|---|
|
BINDER->LIGAND |
BINDING
|
||
|
TARGET -> INDUCER |
Exon 51 skipping. Eteplirsen is a morpholino antisense oligomer which triggers excision of exon 51 during pre-mRNA splicing of the dystrophin RNA transcript.
|
| Related Record | Type | Details | ||
|---|---|---|---|---|
|
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ACTIVE MOIETY |
|
Structural Modifications
| Modification Type | Location Site | Location Type | Residue Modified | Extent | Fragment Name | Fragment Approval |
|---|---|---|---|---|---|---|
| NUCLEOTIDE SUBSTITUTION | [1_1] | SITE_SPECIFIC | CASIMERSEN 5'-CYTOSINE MODIFICATION | 8CQO07490I |