8
Case#: Patient 8, male
DiseaseAssertion: MPS I
FamilyInfo: N/A
CasePresentingHPOs: N/A
CaseHPOFreeText: N/A
CaseNotHPOs: N/A
CaseNotHPOFreeText: N/A
CaseEnzymeAssay: Low IDUA enzyme activity 1.50 (Ref. 4.87 ~ 54.70 μmol/g protein/h)
CaseUrineGAGs: Negative GAG tests
CaseERT: N/A
CaseBMT: N/A
Variant1: c.1081G>A (p.A361T)
Variant1ClinVarID: 92624
Variant1CAID: CA145865
Variant2: c.1395delC (p.G466Afs*?)
Variant2ClinVarID: 929236
Variant2CAID: CA549264595
AdditionalVariants: N/A
ParentalGenotype: N/A
PreviouslyPublished N/A