Fig. 2: Primary endpoint and key functional secondary endpoints.

a, Forest plot showing the primary endpoint (change from baseline to week 52 in NSAA total score, points) and key functional secondary endpoints (change from baseline to week 52 in TTR, seconds, and change from baseline to week 52 in 10MWR, seconds) for delandistrogene moxeparvovec and placebo groups in the modified intent-to-treat population. LSMs (of change from baseline) and CIs were standardized by dividing by the s.e. LSM differences are on original scale (without s.e. adjustment). TTR and 10MWR signs were reversed in the forest plot to align favorable directions among endpoints. Numerical results of LSM difference kept the original signs. One patient in the placebo group had missing data at week 52; functional tests were marked as invalid by the clinical evaluator due to back pain from compression fractures. b, Line graph showing LSM change from baseline to week 52 in NSAA total score, points, for delandistrogene moxeparvovec (n = 63) and placebo (n = 61) groups in the modified intent-to-treat population. Data are presented as LSM values ± 95% CI. c, Line graph showing LSM change from baseline to week 52 in TTR, seconds, for delandistrogene moxeparvovec (n = 63) and placebo (n = 61) groups in the modified intent-to-treat population. Data are presented as LSM values ± 95% CI. d, Line graph showing LSM change from baseline to week 52 in 10MWR, seconds, for delandistrogene moxeparvovec (n = 63) and placebo (n = 61) groups in the modified intent-to-treat population. Data are presented as LSM values ± 95% CI. a–d, The widths of the CIs have not been adjusted for multiplicity and cannot be used to infer definitive treatment effects. Negative values for TFTs (TTR and 10MWR) show an improvement in the time taken to achieve these endpoints.