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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