Survival curves by treatment.
Kaplan–Meier survival curves for the late treatment group (n = 8), early ERT group (n = 6), and early HSCT group (n = 3) of patients are shown. Survival was defined as the period from birth to death; data collection was censored at the final recording point for patients who were alive at the time of this analysis. ERT, enzyme replacement therapy; HSCT, hematopoietic stem cell transplantation.