Evidence to inform the EAG’s preferred comparison was also limited as there was no evidence for the effectiveness of onasemnogene abeparvovec as a treatment for patients with type 1 SMA and three copies of the SMN2 gene, as the START [21], STR1VE-US [22] and STR1VE-EU [23] trials only included patients with type 1 SMA and two copies of the SMN2 gene. The gene discussed is SMN2; the disease is spinal muscular atrophy, type 1.