Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Institute of Medical Genetics and Genomics, |
RCV003234979 | SCV003932873 | likely pathogenic | Niemann-Pick disease, type C1 | 2023-06-21 | criteria provided, single submitter | clinical testing | This heterozygous variantc.3246T>A (p.Ser1082Arg) has been identified in a proband with persistent neonatal cholestatic jaundice in a compound heterozygous state with other variant c.1522G>A (p.Asp508Asn). The Lyso-SM-509 biomarker for Neimann Pick C is increased. This variant is present in exon 22 which is an exonic hotspot and 241 pathogenic mis-sense variants have been reported in this gene (PM1_Moderate & PP2_supporting). This variant is found in 0.0004% gnomAD (aggregated) (PM2_Moderate). |