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Golodirsen

CHEMBL4297762 Phase 4 승인됨 Oligonucleotide
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

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.

작용 기전

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)

기전

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.

자주 묻는 질문

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 section for drug detail pages. Renders standard pharmacological database links plus the drug's data_sources field. #}

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.

의학적 면책조항

This content is for educational and informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before making medication decisions.

Data sources: ChEMBL, PubChem, DailyMed.