Posted by Michael Wonder on 19 Apr 2023
Onasemnogene abeparvovec for the treatment of patients with presymptomatic spinal muscular atrophy
19 April 2023 - NICE has published Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for the treatment of babies aged 12 months and under with presymptomatic spinal muscular atrophy.
Onasemnogene abeparvovec is recommended as an option for the treatment of babies aged 12 months and under with presymptomatic 5q spinal muscular atrophy with a biallelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.
It is only recommended if Novartis provides it according to the commercial arrangement.
