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Nusinersen Sodium

CHEMBL3833342 Phase 4 Đã phê duyệt Oligonucleotide
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

A sodium salt form of nusinersen, an antisense oligonucleotide that increases production of functional SMN protein by modifying splicing of the SMN2 gene, used to treat spinal muscular atrophy (SMA). It is administered by intrathecal injection to reach the spinal cord directly.

Cơ chế tác dụng

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)

Cơ chế

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.

Câu hỏi thường gặp

A sodium salt form of nusinersen, an antisense oligonucleotide that increases production of functional SMN protein by modifying splicing of the SMN2 gene, used to treat spinal muscular atrophy (SMA). It is administered by intrathecal injection to reach the spinal cord directly.

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, Nusinersen Sodium 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). CHEMBL3833342. Open-access bioactivity database.

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

Tuyên bố miễn trách y tế

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.