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Table 8 Change from baseline of liver and spleen volumes 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 liver spleen
baseline 52 weeks baseline 52 weeks
mL %BW mL %BW mL %BW mL %BW
202 445.7 2.27 404.4 1.96 76.3 0.39 87.8 0.43
203 546.1 2.86 518.7 2.44 108.9 0.57 107.3 0.50
204 930.0 2.51 800.9 1.75 149.1 0.40 107.6 0.24
205 691.1 2.80 548.8 1.78 174.0 0.70 107.1 0.35
206 570.2 4.92 486.2 3.33 87.7 0.76 75.5 0.52
207 529.3 2.82 483.7 2.27 209.7 1.12 181.1 0.85
211 528.1 4.63 434.2a 3.37 88.0 0.77 58.1a 0.45
213 1273.5 3.31 1198.4 2.65 210.7 0.55 247.1 0.55
214 601.5 4.63 530.6 3.38 139.0 1.07 118.4 0.75
215 702.6 1.77 765.7 1.50 202.9 0.51 202.5 0.40
216 578.6 2.19 642.5 2.09 168.2 0.64 156.4 0.51
mean 672.4 3.15b 619.5 2.41b 146.8 0.68c 131.7 0.50c
SEM 71.8 0.33 69.8 0.21 15.4 0.07 17.6 0.05
  1. a52-week MRI could not be performed; difference computed from 26-week volume
  2. bLiver %BW at 52 weeks is significantly reduced compared to %BW at baseline (P<0.0005)
  3. cSpleen %BW at 52 weeks is significantly reduced compared to %BW at baseline (P<0.005)
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172