Fig. 2: Part B cohort with infantile-onset SMA: CHOP-INTEND (primary endpoint), NfL levels and EFS.

a, The primary analysis was performed using ranking based on the CHOP-INTEND score at day 183 or day of death and fitting an ANCOVA model adjusting for disease duration and baseline CHOP-INTEND to determine the difference in ranks via the joint-rank test. MI was used to impute missing data. b, Results are from an ANCOVA model with adjustment for participants’ disease duration, baseline log plasma NfL and baseline CHOP-INTEND score. MI was used to impute missing data. In the table, the LSGM ratio from the ANCOVA is shown; the P value was determined using the joint-rank test. c, The P value was determined using the log-rank test stratified according to disease duration (≤12 weeks or >12 weeks). P values were obtained by comparing the two treatment groups indicated per row in each table above each chart. Comparisons between 50/28 mg and 12/12 mg, and between 50/28 mg and matched sham, were performed as separate analyses. In addition to the P value, rankings, changes from baseline in actual scores and ratios are shown. For the EFS and OS, comparisons were similarly made between the two treatment groups. Because of hierarchical testing, none of the time-to-event endpoints were considered statistically significant. Where P < 0.05 but hierarchical testing had stopped, the P value was considered nominally significant and is denoted. ^aP value was considered nominally significant. Study day 1 was the baseline. All statistical tests were two-sided. LSGM, least-squares geometric mean.