U.S. Department of Health & Human Services
National Institutes of Health
NCATS
Return Home
Inxight
Drugs
Search Substances
Home
Browse Drugs
US Approved Drugs
Marketed Drugs
Investigational Drugs
All Substances
Search
Structure Search
Sequence Search
Downloads
About
Search Substances
CASIMERSEN
X8UHF7SX0R
US Approved Rx (2021)
First approved in 2021
General
Record Details
Names
10
Classification
1
Identifiers
9
Subunits
1
Sugars
1
Linkages
1
Relationships
1
Active Moiety
1
Modifications
5
Characteristic Attributes
1
CASIMERSEN
X8UHF7SX0R
US Approved Rx (2021)
First approved in 2021
General
Approval Year
2021
470
Record Details
Substance Class
Nucleic Acid
Nucleic Acid Type
OLIGONUCLEOTIDE
Nucleic Acid Subtype
Other EXON-SKIPPING
Sequence Type
COMPLETE
Record UNII
X8UHF7SX0R
Record Status
Validated (UNII)
Record Version
25
24
23
22
21
20
19
18
17
16
15
14
13
12
11
10
9
8
7
6
5
4
3
2
1
Names
10
Show
5
20
100
All
entries
Name
Type
Language
Name
Type
Language
CASIMERSEN
Official Name
English
CASIMERSEN [USAN]
Common Name
English
EXON-45: NG-12-0064
Code
English
casimersen [INN]
Common Name
English
Casimersen [WHO-DD]
Common Name
English
Showing 1 to 5 of 10 entries
Previous
1
2
Next
Classification
1
Show
5
20
100
All
entries
Classification Tree
Code System
Code
Classification Tree
Code System
Code
Designated/Approved
ORPHAN DRUG
Designated/Approved
Treatment of Duchenne muscular dystrophy
FDA ORPHAN DRUG
690019
Showing 1 to 1 of 1 entries
Previous
1
Next
Identifiers
9
Show
5
20
100
All
entries
Code System
Code
Type
Description
Code System
Code
Type
Description
CAS
1422958-19-7
PRIMARY
RXCUI
2480096
PRIMARY
FDA UNII
X8UHF7SX0R
PRIMARY
WIKIPEDIA
Casimersen
PRIMARY
NCI_THESAURUS
C169830
PRIMARY
Showing 1 to 5 of 9 entries
Previous
1
2
Next
Subunits
1
Subunit 0
>SUBUNIT_0
Sugars
1
Show
5
20
100
All
entries
Sugar
Site Range
Site Count
Sugar
Site Range
Site Count
morpholino
1_1-1_22
22 / 22
Showing 1 to 1 of 1 entries
Previous
1
Next
Linkages
1
Show
5
20
100
All
entries
Linkage
Site Range
Site Count
Linkage
Site Range
Site Count
Phosphoryldimethylamine
1_2-1_22
21 / 21
Showing 1 to 1 of 1 entries
Previous
1
Next
Relationships
1
Show
5
20
100
All
entries
Related Record
Type
Details
Related Record
Type
Details
60RX8439K2
DYSTROPHIN
TARGET -> INDUCER
Comments:
Exon 45 skipping
Showing 1 to 1 of 1 entries
Previous
1
Next
Active Moiety
1
Show
5
20
100
All
entries
Related Record
Type
Details
Related Record
Type
Details
X8UHF7SX0R
CASIMERSEN
ACTIVE MOIETY
Showing 1 to 1 of 1 entries
Previous
1
Next
Modifications
5
Structural Modifications
Show
5
20
100
All
entries
Modification Type
Location Site
Location Type
Residue Modified
Extent
Fragment Name
Fragment Approval
Modification Type
Location Site
Location Type
Residue Modified
Extent
Fragment Name
Fragment Approval
NUCLEOSIDE_SUBSTITUTION
[1_4] [1_9] [1_12] [1_17] [1_18] [1_21]
MORPHOLINO THYMIDINE
YML1Y6H5HD
NUCLEOSIDE_SUBSTITUTION
[1_2] [1_3] [1_8] [1_15]
MORPHOLINO ADENOSINE
3ZYB3DY5HB
NUCLEOSIDE_SUBSTITUTION
[1_5] [1_13] [1_14] [1_16] [1_22]
MORPHOLINO GUANOSINE
XHA1WD2RB2
NUCLEOSIDE_SUBSTITUTION
[1_6] [1_7] [1_10] [1_11] [1_19] [1_20]
MORPHOLINO CYTIDINE
KFE1UKU3TI
NUCLEOTIDE SUBSTITUTION
[1_1]
5'-TERMINUS
CASIMERSEN 5'-CYTOSINE MODIFICATION
8CQO07490I
Showing 1 to 5 of 5 entries
Previous
1
Next
Characteristic Attributes
1
Show
5
20
100
All
entries
Name
Property Type
Amount
Referenced Substance
Defining
Parameters
References
Name
Property Type
Amount
Referenced Substance
Defining
Parameters
References
MOL_WEIGHT:NUMBER(CALCULATED)
CHEMICAL
NUMBER
Showing 1 to 1 of 1 entries
Previous
1
Next
