Skip to main content

Table 4 AES and DQ and Change from Baseline of DQ at week 52 using BSID-III or KABC-II

From: Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial

patient

Test

AES (months)

DQ

Baseline

26 wk

52 wk

Baseline

26 wk

52 wk

52 wk-baseline

202

BSID-III

cognition

0.5

9

6

0.45a

7.9

4.8

4.3

205

19

23

23

31.7

34.9

32.4

0.7

206

25

24

27

67.6

57.1

56.3

-11.3

207

10

16

na

14.1

20.8

na

7.7b

211

10

8

4∙7

33.3

20.0

10.2

-23.1

214

13

16

20

54.2

53.3

55.6

1.4

215

25

26

25

17.2

17.2

16.0

-1.2

216

27

28

28

28.7

28.0

26.4

-2.3

203

KABC-II NVI

59

55

70

80.8

67.9

80.5

-0.3

204

60

66

61

33.9

36.3

32∙5

-1.4

213

65

82

93

34.6

42.3

46.5

11.9

mean

   

36.1

35.1

36.1

-1.2

SEM

   

7.1

5.8

7.4

2.8

  1. na not available
  2. aBaseline DQ not available; 13-week DQ is shown
  3. b52-week DQ not measured; difference computed from 26-week score