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Table 2 Stage 2 Hurler and Hurler-Scheie patients

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 Type Dx age (y) Yrs on ERT Height (cm) Weight (kg) Enroll. Age (y) ID (mg/kg) Genotype
Allele 1 Allele 2
202 H 0.7 8 101 19.6 9.0 1 p.W402X p.W402X
203 H-S 4.9 1.7 104 19.1 6.1 3 p.R621X het IVS9-12-4delCAGGCCCCG
204 H-S 5.0 9 144 37.1 14.8 1 p.R48del p.P533R
205 H 1.1 1.5 112 24.7 5.0 1 c.494-1G>A c.494-1G>A
206a H 1.8 1 81 11.6 3.1 1 p.W402X p.W402X
207 H 2.6 3 95 18.8 5.9 3 p.Q70X p.W402X
211 H 2.4 0 78 11.4 2∙5 3 p.G208D p.G208D
213 H-S 3.7 12 143 38.5 15.7 3 p.R383H p.W402X
214 H 1.8 0 85 13.0 2.0 3 p.W402Term p.W402Term
215 H 3.6 5.6 137 39.6 12.1 6 p.P533R p.G208D
216 H 0.7 5.6 118 26.4 7.8 6 p.G208D p.P533R
  1. apatient had failed bone marrow transplant 8 months prior to enrollment