Disposition of patients in 20 mg/kg qow and 40 mg/kg qow groups.

Mean left ventricular mass Z-scores of treated patients from baseline to week 52; vertical bars represent standard error.

Kaplan-Meier analyses of survival, survival free of invasive ventilation, and survival free of any ventilation. In each panel, thick solid lines show the Kaplan-Meier estimates for the treated patient group; thin solid lines show those for the historical control group, with 95% confidence intervals given by the corresponding dashed lines. Solid circles indicate right-censored observations. (A) Kaplan-Meier estimate of time from date of birth to death. Seven patients were right-censored from this analysis because they had not reached age 36 months by the end of the study, although they remained alive at that time. (B) Kaplan-Meier estimate of time from birth to invasive ventilator use or death. Four patients were right-censored from this analysis because they had not reached age 36 months by the end of the study, although they remained free of invasive ventilation that time. (C) Kaplan-Meier estimate of time from birth to any ventilator use or death. Four patients were right-censored from this analysis because they had not reached age 36 months by the end of the study, although they remained free of any ventilation that time. *Asterisk indicates that 1 patient from the historical control group remained alive at 36 months of age; this patient died at age 44 months.