1
Case#: Patient 1 (see Table 2), female, 18.6 years old (age at report), 5.9 years (years of treatment), 6 surgeries.
DiseaseAssertion: MPS1-HS
FamilyInfo: N/A
CasePresentingHPOs: HP:0002176, HP:0002360 (Spinal cord compression, sleep abnormality)
CaseHPOFreeText: Psychiatric problems
CaseNotHPOs: HP:0000238 (Hydrocephalus)
CaseNotHPOFreeText: Shunt Placement
CaseEnzymeAssay: N/A
CaseUrineGAGs: N/A
CaseERT: Yes
CaseBMT: N/A
Variant1: c.712T>A (p.L238Q) CDNA and protein change do not match. Known variant p.L238Q has a CDNA change of c.713T>A.
Variant1ClinVarID: 265418
Variant1CAID: CA2802065
Variant2: c.63delC (p.P22Rfs*86) Known HGVS nomenclature is c.65del as there is a run of 5 C's.
Variant2ClinVarID: 929238
Variant2CAID: CA437911597
AdditionalVariants: N/A
ParentalGenotype: N/A
PreviouslyPublished N/A