Total submissions: 3
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Counsyl | RCV000669613 | SCV000794384 | uncertain significance | Niemann-Pick disease, type A | 2017-10-07 | criteria provided, single submitter | clinical testing | |
Labcorp Genetics |
RCV002532089 | SCV003439786 | uncertain significance | Niemann-Pick disease, type B; Niemann-Pick disease, type A | 2024-12-09 | criteria provided, single submitter | clinical testing | This sequence change replaces alanine, which is neutral and non-polar, with valine, which is neutral and non-polar, at codon 454 of the SMPD1 protein (p.Ala454Val). This variant is present in population databases (no rsID available, gnomAD 0.006%). This missense change has been observed in individual(s) with clinical features of Niemann-Pick disease (PMID: 12369017). ClinVar contains an entry for this variant (Variation ID: 554055). Invitae Evidence Modeling of protein sequence and biophysical properties (such as structural, functional, and spatial information, amino acid conservation, physicochemical variation, residue mobility, and thermodynamic stability) has been performed for this missense variant. However, the output from this modeling did not meet the statistical confidence thresholds required to predict the impact of this variant on SMPD1 protein function. In summary, the available evidence is currently insufficient to determine the role of this variant in disease. Therefore, it has been classified as a Variant of Uncertain Significance. |
Gene |
RCV003223664 | SCV003919563 | likely pathogenic | not provided | 2022-10-18 | criteria provided, single submitter | clinical testing | Identified in a cohort of patients with Type B Niemann-Pick Disease, although the number of patients harboring this variant (aka A452V) nor clinical details were provided (Simonaro et al., 2002); Not observed at significant frequency in large population cohorts (gnomAD); In silico analysis supports that this missense variant has a deleterious effect on protein structure/function; This variant is associated with the following publications: (PMID: 12369017) |