Our results confirm the efficacy and tolerability of LEV in children with refractory epilepsies and demonstrate good response and retention rates at 12 months. It represents the largest cohort of paediatric patients published so far on LEV with a 1-year follow-up.

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Results Nine children (six girls) had received Nusinersen since June 2017. They had a median (range) age of 22 (9-89) months. The median (range) number of Nusinersen doses was 5 (4-6). Seven children receive BiPAP and one child is ventilated via tracheostomy. The total number of days in hospital since diagnosis was 665 with median (range) hospital days of 64 (4-177) days. This included HDU 52 (4-116) days and PICU 4 (0-103) days. This equates to each child spending a mean of 32% of their life on HDU/PICU. Three spent £2% of their life in hospital, three 30%-60% and three>60%. Conclusion Patients with SMA1 treated with Nusinersen are likely to spend at least a third of their early life in hospital and parents should be counselled accordingly. Treatment with Nusinersen is costly, not only because of the cost of the drug, but because of significant health care utilisation, including time spent receiving critical care.

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