It addresses the genetic cause of SMA by delivering a functional copy of the SMN1 gene into nerve cells. This gene is critical for the function of the nerves that control muscles.
On 7 July 2021 NICE published final guidance for the treatment, making it available to babies with SMA type 1 if they are 6 months or younger; or they are aged 7 to 12 months and their treatment is agreed by the national multidisciplinary team. More information on NICE’s guidance can be found at https://www.nice.org.uk
Zolgensma was accepted for restricted use within NHSScotland by the Scottish Medicines Consortium in March 2021. More information about its assessment can be found at https://www.scottishmedicines.org.uk/medicines-advice/onasemnogene-abeparvovec-zolgensma-full-smc2311/