Extended Data Fig. 3: Part B infantile-onset – Overall survival.

^aIndicates nominal significance; P value is from log-rank test stratified by disease duration ( ≤12 weeks or >12 weeks). All P values were obtained from comparing the two treatment groups indicated per row in each table above each chart. Due to the hierarchical testing, none of the time-to-event endpoints were considered statistically significant. Where a P value was <0.05 but hierarchical testing had stopped, the P value was considered nominally significant and is denoted (a). All statistical tests were two sided. CI, confidence interval; EFS, event-free survival; HR, hazard ratio.