Nusinersen
Nusinersen is an antisense oligonucleotide therapy designed to treat Spinal Muscular Atrophy (SMA), a rare genetic disorder caused by mutations in the SMN1 gene on chromosome 5q that result in insufficient SMN protein production. The medication works by targeting the SMN2 gene to increase inclusion of exon 7, thereby producing more functional SMN protein. It is indicated for use across the spectrum of SMA disease, including pediatric and adult patients.
Nusinersen is an antisense oligonucleotide therapy that increases SMN protein production to treat Spinal Muscular Atrophy caused by chromosome 5q mutations.
Key facts at a glance
- ATC
- M09AX07
- RxNorm
- 1863556
- Generic
- Nusinersen
- Class
- atc-m09
- Brands
- Spinraza
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 12mg
- Maximum
- consult physician
- Frequency
- loading: days 0, 14, 28, 63; maintenance: every 4 months
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 12mg
- Maximum
- consult physician
- Frequency
- loading: days 0, 14, 28, 63; maintenance: every 4 months
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 12mg
- Maximum
- consult physician
- Frequency
- loading: days 0, 14, 28, 63; maintenance: every 4 months
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
10 groupspregnancy
first trimesteravoid if possible
use only if benefits outweigh potential risks to fetus
regular fetal monitoring including ultrasound assessments
Use with caution and monitor for adverse effects.
pregnancy
second trimesteravoid if possible
use only if benefits outweigh potential risks to fetus
regular fetal monitoring including ultrasound assessments
Use with caution and monitor for adverse effects.
pregnancy
third trimesteravoid if possible
use only if benefits outweigh potential risks to fetus
regular fetal monitoring and assessment of fetal growth
Use with caution and monitor for adverse effects.
lactation
breastfeedingno adjustment
discontinue breastfeeding or discontinue drug based on risk-benefit assessment
monitor infant for any adverse effects
pediatric_age
neonates infantsno adjustment
approved for use in pediatric patients including neonates with genetically confirmed SMA
regular neurological assessments and monitoring for respiratory complications
pediatric_age
children adolescentsno adjustment
approved for use in pediatric patients with genetically confirmed SMA
regular neurological assessments and motor function monitoring
geriatric
elderlyuse with caution
limited clinical experience in patients over 65 years; use with close monitoring
monitor renal function and for adverse reactions
Use with caution and monitor for adverse effects.
renal_impairment
mildno adjustment
use with caution; monitor renal function
regular monitoring of renal function
Use with caution and monitor for adverse effects.
renal_impairment
moderate severeadjust dose based on clinical response
use with caution; limited data in severe renal impairment
frequent renal function monitoring
Use with caution and monitor for adverse effects.
hepatic_impairment
mild moderateno adjustment
use with caution; not metabolized by CYP450 enzymes
monitor liver function tests
Use with caution and monitor for adverse effects.
Statistics and clinical data
FDA approval year
FDA approval date for Nusinersen (Spinraza)
Drug class
Pharmacological classification
Mechanism of action
Molecular mechanism of action
Indication
Therapeutic indication
Nusinersen is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients, including those with various disease subtypes ranging from infantile-onset to later-onset forms.
As an antisense oligonucleotide, nusinersen binds to a specific site on the SMN2 pre-messenger RNA, promoting the inclusion of exon 7 during splicing. This results in the production of a more stable and functional SMN protein, compensating for the deficiency caused by SMN1 gene mutations.
Nusinersen is administered via intrathecal injection (into the cerebrospinal fluid) by a healthcare professional. Treatment requires a loading dose schedule followed by maintenance doses. Safety monitoring for thrombocytopenia and renal toxicity is recommended. Patients should be monitored for signs of hyponatremia. The medication should be prescribed only by physicians experienced in managing SMA.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy