CDEK
← Mechanisms of action

Dystrophin pre-mRNA

NUCLEIC-ACIDHomo sapiens

Drugs acting on this target (26)

Active Pharmaceutical Ingredient Mechanism Type ▲ Approval Status API Type Mechanism of Action
golodirsen (Vyondys 53)ActivatorapprovedGene therapyDystrophin pre-mRNA activator
drisapersenAntisense inhibitorexperimentalNucleic acidDystrophin pre-mRNA antisense inhibitor
ns-050/ncnp-03Antisense inhibitorexperimentalNucleic acidDystrophin pre-mRNA antisense inhibitor
pro053Antisense inhibitorexperimentalNucleic acidDystrophin pre-mRNA antisense inhibitor
scaav9.u7.accaAntisense inhibitorexperimentalGene therapyDystrophin pre-mRNA antisense inhibitor
viltolarsen (Viltepso)Antisense inhibitorapprovedNucleic acidDystrophin pre-mRNA antisense inhibitor
wve-n531Antisense inhibitorexperimentalNucleic acidDystrophin pre-mRNA antisense inhibitor
eteplirsen (Exondys 51)ModulatorapprovedNucleic acidDystrophin pre-mRNA modulator
drisapersenOtherexperimentalNucleic acidDystrophin pre-mRNA other
casimersen (Amondys 45)Positive modulatorapprovedGene therapyDystrophin pre-mRNA positive modulator
drisapersenPositive modulatorexperimentalNucleic acidDystrophin pre-mRNA positive modulator
eteplirsen (Exondys 51)Positive modulatorapprovedNucleic acidDystrophin pre-mRNA positive modulator
golodirsen (Vyondys 53)Positive modulatorapprovedGene therapyDystrophin pre-mRNA positive modulator
viltolarsen (Viltepso)Positive modulatorapprovedNucleic acidDystrophin pre-mRNA positive modulator
aoc 1044SupplementationexperimentalGene therapyDystrophin pre-mRNA supplementation
crd-tmh-001SupplementationexperimentalGene therapyDystrophin pre-mRNA supplementation
delandistrogene moxeparvovec (ELEVIDYS)SupplementationapprovedGene therapyDystrophin pre-mRNA supplementation
fordadistrogene movaparvovecSupplementationexperimentalGene therapyDystrophin pre-mRNA supplementation
jwk007SupplementationexperimentalGene therapyDystrophin pre-mRNA supplementation
pgn-edo51SupplementationexperimentalGene therapyDystrophin pre-mRNA supplementation
raav2.5-cmv-minidystrophinSupplementationexperimentalGene therapyDystrophin pre-mRNA supplementation
raavrh74.mck.micro-dystrophinSupplementationexperimentalGene therapyDystrophin pre-mRNA supplementation
rgx-202SupplementationexperimentalGene therapyDystrophin pre-mRNA supplementation
sgt-001SupplementationexperimentalGene therapyDystrophin pre-mRNA supplementation
sgt-003SupplementationexperimentalGene therapyDystrophin pre-mRNA supplementation
sqy51SupplementationexperimentalGene therapyDystrophin pre-mRNA supplementation