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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