Golodirsen
This antisense oligonucleotide therapy is used to treat Duchenne muscular dystrophy (DMD) in patients with a mutation amenable to exon 53 skipping. It works by binding to specific sites on the dystrophin pre-mRNA and redirecting the splicing process to produce a shorter but partially functional dystrophin protein. It is administered by intravenous infusion.
Mecanismo de Ação
Binds to complementary mRNA sequences through Watson-Crick base pairing, modulating gene expression by promoting mRNA degradation via RNase H or altering pre-mRNA splicing.
Pharmacokinetics (PK)
Pharmacodynamics (PD)
Binds to complementary mRNA sequences through Watson-Crick base pairing, modulating gene expression by promoting mRNA degradation via RNase H or altering pre-mRNA splicing.
HBD / HBA
- / -
No targets recorded
Target interaction data is not yet available for this drug.
No interactions recorded
Drug interaction data is not yet available for this compound.
No side effects recorded
Side effect data is not yet available for this drug.
Perguntas frequentes
This antisense oligonucleotide therapy is used to treat Duchenne muscular dystrophy (DMD) in patients with a mutation amenable to exon 53 skipping. It works by binding to specific sites on the dystrophin pre-mRNA and redirecting the splicing process to produce a shorter but partially functional dystrophin protein. It is administered by intravenous infusion.
Binds to complementary mRNA sequences through Watson-Crick base pairing, modulating gene expression by promoting mRNA degradation via RNase H or altering pre-mRNA splicing.
Yes, Golodirsen is an approved drug. It has reached clinical phase 4. It is classified as a Oligonucleotide.
References & Data Sources
- ChEMBL — European Bioinformatics Institute (EBI). CHEMBL4297762. Open-access bioactivity database.
Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.
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