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Page last checked: 29th March 2023

On 8th March 2021, the Scottish Medicines Consortium (SMC) concluded the first Health Technology Appraisal of Zolgensma in Europe and announced its landmark decision giving access to some children who have SMA Type 1.

These children are:

  • infants where both copies of the SMN1 gene are ‘faulty’ and who have a clinical diagnosis of SMA Type 1;
  • pre-symptomatic infants where both copies of the SMN1 gene are ‘faulty’, and up to 3 copies of the SMN2 gene, who are expected to develop SMA Type 1.

All infants are referred to Royal Hospital for Children Glasgow for Zolgensma treatment. Referring centres are:

  • Royal Aberdeen Children’s Hospital
  • Nine Wells Hospital – Dundee
  • Royal Hospital for Sick Children Edinburgh

We understand that Scotland followed NHS England’s 9th January – 28th March temporary pause in eligibility for children older than 12 completed months. This was to investigate and resolve safety concerns – the pause has now been lifted. Read more on this page.

Our summary guide Drug Treatments for Children in England who have SMA may also have useful sections.

Our November 2022 webinar discusses progress with the programme in the UK: