Table 1 Patient baseline and demographic characteristics (safety population)

Age (years)

  Mean (SD)

42.7 (10.6)

46.7 (12.3)

44.0 (11.0)

  Median (range)

44.0

(24, 60)

50.0

(26, 59)

44.5

(24, 60)

Age starting ERT (years)

  Mean (SD)

32.6 (11.8)

39.4 (11.6)

34.8 (11.9)

  Median (range)

37.0

(12, 49)

41.0

(21, 53)

38.0

(12, 53)

% Residual enzyme activity in leukocytes^a

  Mean (SD)

4.8 (2.5)

27.9 (10.2)

12.2 (12.5)

  Median (range)

4.0

(2, 10)

23.7

(16, 46)

5.3

(2, 46)

% Residual enzyme activity in plasma

  Mean (SD)

2.2 (3.2)

28.5 (12.7)

10.6 (14.5)

  Median (range)

0.8

(0.1, 13)

23.9

(17, 51)

2.5

(0.1, 51)

Plasma lyso-Gb₃ (nmol/L)^b

  Mean (SD)

49.7 (45.8)

13.8 (6.1)

38.3 (41.2)

  Median (range)

39.9

(1, 189)

12.9

(7, 23)

27.6

(1, 189)

Plasma Gb₃ (µmol/L)^c

  Mean (SD)

6.3 (2.1)

5.5 (1.9)

6.0 (2.0)

  Median (range)

6.2

(3, 11)

6.1

(3, 8)

6.2

(3, 11)

eGFR_CKD-EPI (mL/min/1.73 m²)

  Mean (SD)

80.8 (26.0)

86.1 (17.8)

82.5 (23.4)

  Median (range)

78.1

(49, 124)

87.7

(55, 109)

87.0

(49, 124)

Annualized eGFR slope^d (mL/min/1.73 m²/year)

  Mean (SD)

 − 5.4 (7.1)

− 5.0 (4.4)

 − 5.3 (6.3)

  Median (range)

 − 4.4

(− 21, 6)

 − 3.7

(− 11, 2)

 − 4.3

(− 21, 6)

UPCR categories

  Severely increased, proteinuria (UPCR > 500 mg/g), n (%)

4 (27)

0

4 (18)

  Moderately increased (UPCR ≥ 150 mg/g and ≤ 500 mg/g), n (%)

1 (7)

2 (29)

3 (14)

Patients treated with ACEi/ARB, n (%)

8 (53.3)

4 (57.1)

12 (54.5)