Children after ZOLGENSMA (onasemnogene abeparvovec-xioi)

The video presents short clips of 46 children treated with Zolgensma. It covers the entire spectrum of SMA (Spinal Muscular Atrophy) types - from type 0 to presymptomatic with 4 copies of SMN2. The video includes children who participated in START, STR1VE-US, STR1VE-EU, SPR1NT and STRONG trials. Some of the children in the video have only been on a single treatment, some switched from Spinraza to Zolgensma, while others combined Zolgensma with Spinraza or Evrysdi (Risdiplam) Disclaimer The video represents self-reported segments by families of children treated with Zolgensma. This is entirely a patient-family driven private initiative from the conceptualisation, compilation, editing to publication. No pharmaceutical company was involved in the production of the video at any stage and is not responsible for any of the contents. Moreover, just to make it clear, each family who has contributed to the video has done so in their individual capacity only and are not representing their employers or anyone