Trade Name(s)

  • Spinraza

Ther. Class.
none assigned

Pharm. Class.
temporary class antisense oligonucleotides


Treatment of spinal muscular atrophy.


Increases exon 7 inclusion in survival motor neuron-2 messenger ribonucleic acid (mRNA) transcripts which results in increased production of full-length survival motor neuron protein.

Therapeutic Effect(s):

Reduction in risk of death or permanent ventilation and improvement in motor skills.


Absorption: Delivered via intrathecal injection into cerebrospinal fluid (CSF).

Distribution: Distributed within CNS and peripheral tissues (skeletal muscle, liver, and kidney).

Metabolism and Excretion: Metabolized via exonuclease (3'- and 5')-mediated hydrolysis; primarily excreted in urine.

Half-life: 135–177 days (CSF); 63–87 days (plasma).

TIME/ACTION PROFILE (plasma concentrations)

ITunknown1.7–6 hrunknown


Contraindicated in:

  • None.

Use Cautiously in:

OB:  Lactation: Safety not established

Adverse Reactions/Side Effects

CNS: headache

Derm: rash

EENT: ear infection

GI: constipation, vomiting, flatulence

GU: GLOMERULONEPHRITIS, urinary tract infection

Hemat: BLEEDING, thrombocytopenia

Metabolic: ↓ weight

MS: back pain

Resp: lower respiratory tract infection

Misc: fever

* CAPITALS indicate life-threatening.
Underline indicate most frequent.



None noted.


IT (Adults and Children):  Loading dose– 12 mg every 14 days for 3 doses; then 12 mg given 30 days after the third dose;  Maintenance dose– 12 mg every 4 mo.


Solution for intrathecal injection: 2.4 mg/mL


  • Assess motor development prior to and periodically during therapy.

Lab Test Considerations: May cause bleeding. Monitor platelet count, prothrombin time, and activated partial thromboplastin time at baseline, prior to each injection, and as needed.

  • May cause renal toxicity. Monitor quantitative spot urine protein testing at baseline, prior to each injection, and as needed. For urinary protein concentration >0.2 g/L, consider repeat testing and further evaluation.

Potential Diagnoses


  • Consider sedation during procedure if needed.
  • IT Allow solution to come reach room temperature before administering; do not use external sources of heat. Solution is clear and colorless; do not administer solutions that are discolored or contain particulate matter. Withdraw 12 mg (5 mL) of nusinersen from vial into syringe; discard unused solution in vial. Consider ultrasound or other imaging techniques to guide intrathecal administration, particularly in younger patients. Remove 5 mL cerebrospinal fluid prior to administration. Solution is stable if refrigerated for up to 14 days; protect from light.
  • Rate: Administer as an intrathecal bolus injection over 1–3 min. Do not administer in areas of skin infection or inflammation.

Patient/Family Teaching

  • Explain purpose of nusinersen to patient and caregiver. Emphasize importance of dosing schedule. If a loading dose is delayed or missed, administer dose as soon as possible, with at least 14 days between doses and continue dosing as prescribed. If a maintenance dose is delayed or missed, administer dose as soon as possible and continue dosing every 4 months.
  • Advise patient and caregiver to notify health care professional immediately if signs and symptoms of bleeding (unexpected bleeding, blood in stools or urine) or renal toxicity (edema, dyspnea) occur.

Evaluation/Desired Outcomes

Reduction in risk of death or permanent ventilation and improvement in motor skills.

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Anesthesia Central is an all-in-one web and mobile solution for treating patients before, during, and after surgery. This collection of drug, procedures and test information is derived from Davis’s Drug, MGH Clinical Anesthesia Procedures, Pocket Guide to Diagnostic Tests, and MEDLINE Journals. .