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Medically reviewed on Nov 15, 2018


(nue si NER sen)

Dosage Forms

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Solution, Intrathecal [preservative free]:

Spinraza: 12 mg/5 mL (5 mL)

Brand Names: U.S.

  • Spinraza

Pharmacologic Category

  • Antisense Oligonucleotide


Treats spinal muscular atrophy caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency by binding to a specific sequence in the intron downstream of exon 7 of the SMN2 messenger ribonucleic acid (mRNA) transcript and increase production of full-length SMN protein.


Distributed within the CNS and peripheral tissues


Via exonuclease (3’- and 5’)-mediated hydrolysis



Time to Peak

Median range: 1.7 to 6 hours

Half-Life Elimination

Terminal (mean range): CSF: 135 to 177 days; Plasma: 63 to 87 days

Use: Labeled Indications

Spinal muscular atrophy: Treatment of spinal muscular atrophy (SMA)


There are no contraindications listed in the manufacturer's labeling.

Canadian labeling: Additional contraindications (not in US labeling): Known or suspected hypersensitivity to nusinersen or any component of the formulation.

Dosing: Adult

Spinal muscular atrophy: Intrathecal: Loading dose: 12 mg once every 14 days for 3 doses; then 12 mg once 30 days after the third dose. Maintenance: 12 mg once every 4 months

Dosing: Pediatric

Spinal muscular atrophy: Intrathecal: Neonates, Infants, Children, and Adolescents: Refer to adult dosing.

Dosing: Renal Impairment

There are no dosage adjustments provided in the manufacturer’s labeling.

Dosing: Hepatic Impairment

There are no dosage adjustments provided in the manufacturer’s labeling.


For intrathecal use only. Allow vial to warm to room temperature before use; do not use external heat sources. Prior to administration, remove 5 mL of cerebrospinal fluid. Administer as an intrathecal bolus injection over 1 to 3 minutes. Do not administer in areas with signs of infection or inflammation.


Store intact vials between 2°C to 8°C (36°F to 46°F) in original carton, protected from light. Do not freeze. May store intact vials at or below 30°C (86°F) for up to 14 days in original carton, protected from light. Intact vials in original carton may be removed from and returned to the refrigerator, if necessary; if removed from the original carton, the total combined time out of refrigeration should not exceed 30 hours at a temperature not exceeding 25°C (77°F). Following removal from vial, administer solution within 4 hours; discard unused contents of the vial.

Drug Interactions

There are no known significant interactions.

Adverse Reactions


Central nervous system: Headache (29%)

Gastrointestinal: Constipation (35%), vomiting (29%), period of tooth development (18%)

Genitourinary: Proteinuria (58%)

Hematologic & oncologic: Thrombocytopenia (16%)

Neuromuscular & skeletal: Back pain (25%)

Respiratory: Lower respiratory tract infection (55%), atelectasis (18%)

Miscellaneous: Fever (43%)

1% to 10%:

Central nervous system: Fall (5%)

Endocrine & metabolic: Weight loss (5%)

Gastrointestinal: Flatulence (5%)

Genitourinary: Urinary tract infection (9%)

Hypersensitivity: Seasonal allergy (5%)

Immunologic: Antibody development (6%)

Otic: Otic infection (6%)

Respiratory: Respiratory congestion (5%; upper tract: 8%), epistaxis (7%)

Frequency not defined: Miscellaneous: Postoperative complication (post lumbar puncture syndrome)

<1%, postmarketing, and/or case reports: Hydrocephalus, maculopapular rash, meningitis, serious infection, skin rash


Concerns related to adverse effects:

• Hematologic effects: Coagulation abnormalities and thrombocytopenia (including acute severe thrombocytopenia), have been observed with some antisense oligonucleotides; increased risk of bleeding complications may occur. Perform a platelet count and coagulation testing at baseline, prior to each dose and as clinically needed.

• Renal toxicity: Renal toxicity, including potentially fatal glomerulonephritis, has been observed with some antisense oligonucleotides. Conduct quantitative spot urine protein testing (preferably using first morning urine) at baseline and prior to each dose. For urinary protein concentration >0.2 g/L, consider repeat testing and further evaluation.

Monitoring Parameters

Platelet count, coagulation tests (prothrombin time; activated partial thromboplastin time), and quantitative spot urine protein testing at baseline, prior to each dose, and as clinically indicated.

Pregnancy Considerations

Adverse events have been observed in some animal reproduction studies.

Patient Education

• Discuss specific use of drug and side effects with patient as it relates to treatment. (HCAHPS: During this hospital stay, were you given any medicine that you had not taken before? Before giving you any new medicine, how often did hospital staff tell you what the medicine was for? How often did hospital staff describe possible side effects in a way you could understand?)

• Patient may experience common cold symptoms, constipation, flatulence, weight loss, vomiting, or back pain. Have patient report immediately to prescriber signs of kidney problems (urinary retention, hematuria, change in amount of urine passed, or weight gain), signs of bleeding (vomiting blood or vomit that looks like coffee grounds; coughing up blood; hematuria; black, red, or tarry stools; bleeding from the gums; abnormal vaginal bleeding; bruises without a reason or that get bigger; or any severe or persistent bleeding), signs of a urinary tract infection (hematuria, burning or painful urination, polyuria, fever, lower abdominal pain, or pelvic pain), falls, chills, sputum discoloration, cough, ear pain, or severe headache (HCAHPS).

• Educate patient about signs of a significant reaction (eg, wheezing; chest tightness; fever; itching; bad cough; blue skin color; seizures; or swelling of face, lips, tongue, or throat). Note: This is not a comprehensive list of all side effects. Patient should consult prescriber for additional questions.

Intended Use and Disclaimer: Should not be printed and given to patients. This information is intended to serve as a concise initial reference for health care professionals to use when discussing medications with a patient. You must ultimately rely on your own discretion, experience, and judgment in diagnosing, treating, and advising patients.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.