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Page last checked: 22nd November 2022
Last updated: November 2021


This global trial started in September 2021 is investigating the safety, tolerability and efficacy of intravenous Zolgensma™ in children who have SMA and weigh between 8.5 kgs and 21 kgs. It aims to recruit 24 participants in total across sites in Europe, North America, Australia and Taiwan, and will follow patients for a period of 12 months. UK trial Centres are in London and Newcastle. All currently available information, including inclusion and exclusion criteria, can be found at

In our submissions to NICE, we raised that clinical experts expect that this treatment could also provide health benefits for a proportion of children with SMA Type 2 and 3, and that these health gains would be highly valued by those children and their parents /carers. We acknowledged that no evidence or modelling was presented to NICE to support this and urged Novartis to continue to work to address this gap in access to the treatment. This initiative begins to address this.