Daily Medication Pearl: Nusinersen (Spinraza) for Spinal Muscular Atrophy


Nusinersen (Spinraza) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

Medication Pearl of the Day: Nusinersen (Spinraza)

Indication: Nusinersen is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.


  • Dosing: The recommended dosage is 12 mg (5 mL) per administration
    • Initiate nusinersen treatment with 4 loading doses:
      • The first 3 loading doses should be administered at 14-day intervals.
      • The fourth loading dose should be administered 30 days after the 3 round dose.
      • A maintenance dose should be administered once every 4 months thereafter.
  • Dosage form: Injection: 12 mg/5 mL (2.4 mg/mL) in a single-dose vial.
  • Adverse events: The most common adverse reactions were lower respiratory infection and constipation, pyrexia, headache, vomiting, and back pain in patients.
  • Mechanism of Action: Nusinersen is designed to treat SMA, which caused by mutations in chromosome 5q that lead to SMN protein deficiency. Nusinersen was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid transcripts and production of full-length SMN protein.
  • Manufacturer: Biogen


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