Drug reference
nusinersen
For healthcare professionals — reference only. This page is a brief summary, not medical advice and not a substitute for official labeling or clinical judgement. Always verify before prescribing or dispensing.
Drug class
Central Nervous System (Miscellaneous(CNS))
Mechanism of action
An antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency
Using in vitro assays and studies in transgenic animal models of SMA, nusinersen was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein
Common side effects
- Constipation
- Headache
- Back pain
- Thrombocytopenia
- Lower respiratory tract infections
- Upper respiratory infection
- Atelectasis
- Postlumbar puncture syndrome
- Elevated urine protein
Contraindications
- None
Related conditions
Frequently asked questions
What drug class is nusinersen?
nusinersen belongs to: Central Nervous System.
How does nusinersen work?
An antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency Using in vitro assays and studies in transgenic animal models of SMA, nusinersen was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein
What are the common side effects of nusinersen?
Constipation, Headache, Back pain, Thrombocytopenia, Lower respiratory tract infections, Upper respiratory infection, Atelectasis, Postlumbar puncture syndrome, Elevated urine protein
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