This includes open-label, phase 3, single-arm, single-dose, multi-center trials (STR1VE-US in the United States, STR1VE-AP in Asia Pacific and STR1VE-EU in Europe) designed to assess safety and efficacy in symptomatic patients with SMA type I < six months of age with one or two copies of the SMN2 gene. This evidence concerns the gene SMN2 and spinal muscular atrophy, type 1.