DYSTROPHIN

Investigational

Approval Year

Substance Class	Protein Created by `admin` on `Wed Apr 02 02:13:19 GMT 2025` Edited by `admin` on `Wed Apr 02 02:13:19 GMT 2025`
Protein Type	STRUCTURAL PROTEIN
Protein Sub Type
Sequence Origin	HUMAN
Sequence Type	COMPLETE
Record UNII	60RX8439K2
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

DMD PROTEIN

Preferred Name

English

DYSTROPHIN

Common Name

English

DUCHENNE MUSCULAR DYSTROPHY PROTEIN

Common Name

English

Code System Code Type Description

UNIPROT

P11532

Created by admin on Wed Apr 02 02:13:19 GMT 2025 , Edited by admin on Wed Apr 02 02:13:19 GMT 2025

PRIMARY

FDA UNII

60RX8439K2

Created by admin on Wed Apr 02 02:13:19 GMT 2025 , Edited by admin on Wed Apr 02 02:13:19 GMT 2025

PRIMARY

Linkage Type	Residue Index
ZZ-type Zinc finger (C-x(2)-C-x(4,8)-[RHDGSCV]-[YWFMVIL]-x-[CS]-x(2,5)-[CHEQ]- x-[DNSAGE]-[YFVLI]-x-[LIVFM]-C-x(2)-C)	1_3313	1_3316	1_3337	1_3340

Related Record Type Details


					4KM8P975UM

Brogidirsen

ACTIVATOR OF EXPRESSION->TARGET

Comments:

Dystrophin expression stimulants

Amount:


					033072U4MZ

GOLODIRSEN

INDUCER -> TARGET

Comments:

exon 53 skipping


					X8UHF7SX0R

CASIMERSEN

INDUCER -> TARGET

Comments:

Exon 45 skipping


					73D586DAMF

DRISAPERSEN

INDUCER -> TARGET

Comments:

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.


					34LVJ6J2YV

Nivudirsen

CORRECTOR->TARGET

Comments:

BMN 351-induced exon 51 skips allow for restoration of the mutated open-reading frame and result in significant levels of near full-length dystrophin

Amount:


					AIW6036FAS

ETEPLIRSEN

INDUCER -> TARGET

Comments:

Exon 51 skipping. Eteplirsen is a morpholino antisense oligomer which triggers excision of exon 51 during pre-mRNA splicing of the dystrophin RNA transcript.

Amount:


					NVQ25YNH8P

Pivodisgene Parvec

MIMETIC->TARGET

Amount:


					SXA7YP6EKX

VILTOLARSEN

ACTIVATOR OF EXPRESSION->TARGET

Amount:

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