nusinersen sodium at a glance
Medicine class
Antisense oligonucleotide
Licensed use
Treatment of 5q spinal muscular atrophy
How it is given
Injection into cerebrospinal fluid through a lumbar puncture
Treatment setting
Given by a specialist team experienced in spinal muscular atrophy and lumbar puncture
What is nusinersen sodium used for?
The selected UK product is licensed for:
Treatment of 5q spinal muscular atrophy, including symptomatic and genetically diagnosed presymptomatic disease where specialist assessment supports treatment.
How does nusinersen sodium work?
Nusinersen binds to SMN2 pre-mRNA and promotes inclusion of exon 7. This allows SMN2 to produce more full-length SMN protein needed by motor neurons.
nusinersen sodium preparations and strengths
Injection
Route: Parenteral
Strengths: 12 mg, 28 mg, 50 mg
How to take nusinersen sodium
Nusinersen is not taken by mouth. A trained healthcare professional injects it into the fluid surrounding the spinal cord through a lumbar puncture. Sedation or imaging guidance may be used when clinically appropriate, particularly in young people or those with scoliosis.
Attend the specialist centre as arranged and follow its preparation and aftercare instructions.
Tell the team about illness, skin infection near the planned injection site, allergies and all medicines being used.
Report troublesome or unusual symptoms following the lumbar puncture.
nusinersen sodium side effects
Common or expected effects
- Headache, often related to lumbar puncture
- Vomiting
- Back pain or discomfort after lumbar puncture
- Fever
Get urgent medical advice
- Hydrocephalus
- Meningitis or aseptic meningitis
- Arachnoiditis
- Hypersensitivity reactions, including facial or tongue swelling, hives, rash or itching
- Serious infection associated with lumbar puncture
nusinersen sodium in pregnancy
Human pregnancy data are absent or limited. Product information advises avoiding nusinersen during pregnancy as a precaution; discuss the benefits and uncertainties with the specialist team before treatment.
nusinersen sodium while breastfeeding
It is unknown whether nusinersen or its metabolites enter human milk, and risk to a breastfed infant cannot be excluded. The specialist and parent should weigh the benefit of breastfeeding against the benefit of treatment when deciding whether breastfeeding or treatment should be paused.
nusinersen sodium interactions
Formal interaction studies have not been performed, although laboratory findings suggest a low potential for common metabolic or transporter-mediated interactions.
Medicines metabolised through cytochrome P450 enzymes
A clinically important metabolic interaction is considered unlikely, but the specialist team should review all medicines.
Common questions about nusinersen sodium
Answers are fully visible for fast scanning and source review.
Is nusinersen sodium the same as Spinraza?
Nusinersen sodium is the active ingredient; Spinraza is its brand name.
Can nusinersen sodium cure spinal muscular atrophy?
No. It targets an underlying cause of 5q spinal muscular atrophy but does not remove the genetic condition.
Can adults receive nusinersen sodium?
The licence for 5q spinal muscular atrophy is not restricted to children. Specialist assessment is required, and adult responses vary.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.