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Table 7 Change from baseline in total grey matter volume 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

patient age Total grey matter volume (mm3)
BL 26 wk 52 wk 52wk-BL
202 9.0 797,018 792,018 788,648 -8,370
203 6.1 747,735 768,848 nd +21,113a
204 14.8 517,491 604,125 616,132 +98,641b
205 5.0 710,691 759,444 749,177 +38,486
206 3.1 621,494 673,817 703,973 +82,478
207 5.9 607,678 590,810 598,460 -9,218
211 2.5 589,439 npc npc  
213 15.7 874,710 885,006 863,862 -10,848
214 2.0 744,555 764,571 780,779 +36,224
215 12.1 668,638 663,441 676,069 +7,431
216 7.8 739,492 708,228 717,836 -21,656
mean   692,631    +15,071
SEM   34,396    11,052
  1. BL baseline, nd volumetrics analysis could not be performed
  2. a52-week volumetrics not available; difference computed from 26-week volume
  3. bPatient with braces; volumes not used to compute mean difference at 52 weeks
  4. cnp=not performed; volumetric analysis could not be completed