Spinal muscular atrophy
Treatment of Spinal muscular atrophy
Restricted to patients with symptomatic type 1 SMA (infantile onset); treatment with nusinersen for patients with types 2 and 3 SMA is under the SMC ultra-orphan pathway.
Nusinersen
Nusinersen 12mg/5ml solution for injection vials
Dose as per specialist.
Prescribing Notes:
- Treatment of spinal muscular atrophy should be initiated only on specialist advice.
- Clinical diagnosis of SMA 1 should prompt urgent treatment discussions.
- Nusinersen is SMC approved (1318/18) for treatment of 5q spinal muscular atrophy (SMA); restricted to patients with symptomatic type 1 SMA (infantile onset); treatment with nusinersen for patients with types 2 and 3 SMA is under the SMC ultra-orphan pathway.
History Notes
09/11/2023
East Region Formulary content agreed.