Mean ± SD change over time in plasma/urine Gb
in the transition safety population. (a) Plasma Gb3 (nmol/g creatinine) and (b) urine Gb3 (nmol/mL); the dotted lines represent the annualized slope. Baseline mean ± SD plasma Gb3 and urine Gb3 values at the beginning of phase 1 were 3.61 ± 1.60 nmol/mL and 538.09 ± 681.27 nmol/g creatinine, respectively (both measured at study TKT023 week 25/26). Agalα, agalsidase alfa; CI, confidence interval; Gb3, globotriaosylceramide; SD, standard deviation; yr, year.