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Viltolarsen

CHEMBL4298062 Phase 4 已批准 Oligonucleotide
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

Viltolarsen is a phosphorodiamidate morpholino oligonucleotide (PMO) antisense therapy that binds to exon 53 of dystrophin pre-mRNA, causing exon 53 skipping during splicing to restore the reading frame and enable production of a truncated but partially functional dystrophin protein. It is approved for Duchenne muscular dystrophy (DMD) in patients with confirmed mutations amenable to exon 53 skipping. By restoring a partially functional dystrophin, it slows disease progression in this subset of DMD patients.

作用机制

Delivers synthetic messenger RNA encoding a specific protein to host cells, where it is translated by ribosomes to produce the therapeutic protein without integrating into the host genome.

Pharmacokinetics (PK)

Pharmacodynamics (PD)

机制

Delivers synthetic messenger RNA encoding a specific protein to host cells, where it is translated by ribosomes to produce the therapeutic protein without integrating into the host genome.

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.

常见问题

Viltolarsen is a phosphorodiamidate morpholino oligonucleotide (PMO) antisense therapy that binds to exon 53 of dystrophin pre-mRNA, causing exon 53 skipping during splicing to restore the reading frame and enable production of a truncated but partially functional dystrophin protein. It is approved for Duchenne muscular dystrophy (DMD) in patients with confirmed mutations amenable to exon 53 skipping. By restoring a partially functional dystrophin, it slows disease progression in this subset of DMD patients.

Delivers synthetic messenger RNA encoding a specific protein to host cells, where it is translated by ribosomes to produce the therapeutic protein without integrating into the host genome.

Yes, Viltolarsen 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). CHEMBL4298062. Open-access bioactivity database.

Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.

医疗免责声明

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.