Scotland – Access to Zolgensma
Scotland – Access to Zolgensma
Page last checked: 18th December 2024
On 8th March 2021, the Scottish Medicines Consortium (SMC) completed the first Health Technology Appraisal of Zolgensma in Europe. It announced its landmark decision giving access to Zolgensma for.
- infants who have had a confirmed diagnosis of 5q autosomal recessive SMA – an SMN1 gene deletion test and a clinical diagnosis of SMA Type 1.
- pre-symptomatic infants , who have had a confirmed diagnosis of 5q autosomal recessive SMA – an SMN1 gene deletion test. They must have up to 3 copies of the SMN2 gene and be 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
Our summary guide discussing all the potential treatments may be useful: Drug Treatments for Children who have 5qSMA.
Our Conversations with Health Professionals webinars include latest updates on treatment.