Page last checked: 9th December 2024

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