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