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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)