Nusinersen Sodium
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.
Mécanisme d'action
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.
Foire aux questions
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
- ChEMBL — European Bioinformatics Institute (EBI). CHEMBL3833342. Open-access bioactivity database.
Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.
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