The Scottish Medicines Consortium has accepted nusinersen for restricted use within NHS Scotland for babies with genetically confirmed 5q spinal muscular atrophy before symptoms appear. The decision, published in August 2026, provides clinicians with an additional early treatment option for infants identified through newborn screening or targeted genetic testing.
The SMC advice confirms that nusinersen can be used in pre symptomatic infants with either two or three copies of the SMN2 gene. Evidence reviewed by the committee showed that early treatment was associated with substantially improved outcomes compared with historical cohorts. Most infants treated before symptom onset achieved major motor milestones, including sitting and walking independently, and the majority did not require respiratory support during follow up. The committee concluded that initiating therapy at the earliest possible stage offered clear clinical benefit.
With this decision, all three licensed SMA treatments are now available in Scotland for babies before symptoms develop. The SMC noted that early access enables clinicians and families to make informed choices about therapy based on clinical characteristics, family preference and the evolving evidence base. The advice also highlighted the importance of newborn screening pathways in ensuring timely diagnosis and referral to specialist neuromuscular teams.
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Nusinersen will continue to be prescribed and monitored within specialist services, with ongoing assessment of clinical response. The SMC’s acceptance is expected to support more consistent access to early treatment and may contribute to improved long term outcomes for infants diagnosed through Scotland’s screening programme.
This news item was drafted with AI assistance and reviewed by ACNR’s editorial team.
Sources
https://www.scottishmedicines.org.uk
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