Nusinersen
Spinraza (nusinersen) is a prescription medication used to treat spinal muscular atrophy (SMA), a genetic disorder that affects the nervous system and causes progressive muscle weakness. It is administered directly into the cerebrospinal fluid via intrathecal injection by a healthcare professional.
Spinraza is a treatment for spinal muscular atrophy that works by increasing production of a critical protein deficient in this genetic condition.
Key facts at a glance
- RxNorm
- 1863561
- Generic
- Spinraza
- Class
- brand-name
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- 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.
Spinraza is approved for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients across various subtypes of the disease.
Spinraza is an antisense oligonucleotide that modifies the splicing of the SMN2 gene, enabling increased production of functional survival motor neuron (SMN) protein, which is deficient in patients with SMA.
Spinraza is administered via intrathecal injection by a qualified healthcare professional. Treatment requires a loading dose schedule followed by maintenance doses. Common side effects may include respiratory infections and constipation. Patients should be monitored for low platelet counts and kidney abnormalities during treatment.
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 August 9, 2026. · Read our editorial policy