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Fig. 2 | Orphanet Journal of Rare Diseases

Fig. 2

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

Fig. 2

Concentration of heparan sulfate (HS, panel a) and dermatan sulfate (DS, panel b) in CSF of Stage 2 patients at baseline and at 26 weeks (wks) of treatment in comparison to HS and DS levels in CSF of 10 non-MPSI human pediatric subjects. The mean ± SE values for HS in CSF are 685 ± 112, 779 ± 78, and 91 ± 18 ng/mL for the MPSI patients at baseline, for the MPSI patients at 26 weeks, and for the pediatric controls, respectively. The mean ± SE values for DS in CSF are 436 ± 85, 490 ± 87, and 38 ± 8 ng/mL for the MPSI patients at baseline, for the MPSI patients at 26 weeks, and for the pediatric controls, respectively

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172