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Table 6 Change from Baseline of DQ and AES using non-cognitive domains of BSID-III 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

Receptive communication

13.8 ± 3.7

+1.5 ± 5.2

7.8 ± 2.4

+3.2 ± 2.4

Expressive communication

17.6 ± 4.0

+1.6 ± 3.1

9.3 ± 2.2

+4.3 ± 1.3

Fine motor

34.7 ± 7.7

-5.1 ± 5.1

19.8 ± 4.5

+0.1 ± 3.3

Gross motor

25.6 ± 6.2

-2.3 ± 2.0

13.4 ± 1.8

+1.8 ± 1.1

  1. Mean ± SEM (N=8)