Casimersen
An antisense oligonucleotide that targets a specific sequence in pre-messenger RNA to promote skipping of exon 45 of the dystrophin gene, allowing production of a truncated but partially functional dystrophin protein. It is approved for the treatment of Duchenne muscular dystrophy in patients with mutations amenable to this particular exon skipping. Treatment may slow disease progression in eligible patients.
Etki Mekanizması
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.
Sıkça Sorulan Sorular
An antisense oligonucleotide that targets a specific sequence in pre-messenger RNA to promote skipping of exon 45 of the dystrophin gene, allowing production of a truncated but partially functional dystrophin protein. It is approved for the treatment of Duchenne muscular dystrophy in patients with mutations amenable to this particular exon skipping. Treatment may slow disease progression in eligible patients.
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, Casimersen is an approved drug. It has reached clinical phase 4. It is classified as a Oligonucleotide.
References & Data Sources
- ChEMBL — European Bioinformatics Institute (EBI). CHEMBL4297566. Open-access bioactivity database.
Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.
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