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Antisense oligonucleotide (SMN2 splicing modifier)

Nusinersen

Brand names: Spinraza

Adult dose

Dose: 12mg intrathecal: loading on days 0, 14, 28, 63, then maintenance every 4 months
Route: Intrathecal
Frequency: per protocol

Clinical pearls

  • Spinal muscular atrophy (SMA) — specialist neuromuscular MDT
  • Coag and platelet check before each dose

Contraindications

  • Hypersensitivity

Side effects

  • Post-LP syndrome
  • Thrombocytopenia / coagulopathy
  • Renal toxicity
  • Hydrocephalus (rare)
  • Headache

Monitoring

  • Platelets
  • Coag
  • Urinary protein
  • Neurodevelopment

Reference: BNF; NICE TA588; SmPC; https://bnf.nice.org.uk/drugs/nusinersen/. Verify against your local formulary and the latest BNF before prescribing.

Related

Curated clinical cross-links plus same-class fallbacks.