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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)