Skip to main content

Table 5 Change from Baseline of DQ and AES using VABS-II at week 52

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

domain

DQ

AES (months)

baseline

52 wk-baseline

baseline

52 wk-baseline

Overall

44.4 ± 6.6

+0.6 ± 5.3

37.4 ± 10.6

+12.2 ± 8.0

Communication

42.5 ± 7.7

-1.8 ± 3.8

38.3 ± 15.0

+7.9 ± 5.1

Daily Living Skills

46.7 ± 8.4

-2.5 ± 6.0

38.2 ± 9.3

+7.4 ± 4.5

Socialization

38.7 ± 5.8

+9.5 ± 9.0

32.1 ± 8.8

+21.3 ± 16.5

Motor Ability

43.5 ± 8.2

+1.6 ± 4.6

31.6 ± 6.2

+8.4 ± 3.7

  1. Mean ± SEM (N=11)