U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Approval Year

Substance Class Protein
Created
by admin
on Sat Dec 16 14:56:52 GMT 2023
Edited
by admin
on Sat Dec 16 14:56:52 GMT 2023
Protein Type STRUCTURAL PROTEIN
Protein Sub Type
Sequence Origin HUMAN
Sequence Type COMPLETE
Record UNII
60RX8439K2
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
DYSTROPHIN
Common Name English
DMD PROTEIN
Common Name English
DUCHENNE MUSCULAR DYSTROPHY PROTEIN
Common Name English
Code System Code Type Description
UNIPROT
P11532
Created by admin on Sat Dec 16 14:56:53 GMT 2023 , Edited by admin on Sat Dec 16 14:56:53 GMT 2023
PRIMARY
FDA UNII
60RX8439K2
Created by admin on Sat Dec 16 14:56:53 GMT 2023 , Edited by admin on Sat Dec 16 14:56:53 GMT 2023
PRIMARY
Related Record Type Details
ACTIVATOR OF EXPRESSION->TARGET
Dystrophin expression stimulants
INDUCER -> TARGET
exon 53 skipping
INDUCER -> TARGET
Exon 45 skipping
INDUCER -> TARGET
Completed Phase III trials and did not meet its primary endpoint. Alters the splicing of the dystrophin RNA transcript, eliminating exon 51 from the mature dystrophin mRNA.
INDUCER -> TARGET
Exon 51 skipping. Eteplirsen is a morpholino antisense oligomer which triggers excision of exon 51 during pre-mRNA splicing of the dystrophin RNA transcript.
ACTIVATOR OF EXPRESSION->TARGET
Name Property Type Amount Referenced Substance Defining Parameters References
MOL_WEIGHT:NUMBER(CALCULATED) CHEMICAL