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Table 1 Stage 1 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 Age (years) Genotype
Allele 1 Allele 2
101 28 p.R89Q p.W402X
102 37 p.Q70X p.D445del
103 19 IVS7+1G>A p.X654R
104 31 c.590-6ins4 p.Y343X
105 30 p.R383H p.W402X
106 24 p.R383H p.W402X