Total submissions: 3
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Labcorp Genetics |
RCV003555901 | SCV004293270 | pathogenic | not provided | 2023-10-17 | criteria provided, single submitter | clinical testing | This sequence change creates a premature translational stop signal (p.Trp23*) in the NEU1 gene. It is expected to result in an absent or disrupted protein product. Loss-of-function variants in NEU1 are known to be pathogenic (PMID: 11063730, 14517945). This variant is not present in population databases (gnomAD no frequency). This premature translational stop signal has been observed in individual(s) with sialidosis (PMID: 14695530). ClinVar contains an entry for this variant (Variation ID: 2458). For these reasons, this variant has been classified as Pathogenic. |
OMIM | RCV000002562 | SCV000022720 | pathogenic | Sialidosis type 2 | 2004-01-01 | no assertion criteria provided | literature only | |
Prevention |
RCV003964788 | SCV004779163 | pathogenic | NEU1-related disorder | 2024-02-29 | no assertion criteria provided | clinical testing | The NEU1 c.69G>A variant is predicted to result in premature protein termination (p.Trp23*). This variant has been reported in the homozygous state in two unrelated individuals with type II sialidosis (Patients C and D, Pattison et al. 2004. PubMed ID: 14695530). Of note, these patients had clinically severe phenotypes and both passed away by 2-3 months of age. This variant has not been reported in a large population database (http://gnomad.broadinstitute.org), indicating this variant is rare. Nonsense variants in NEU1 are expected to be pathogenic. This variant is interpreted as pathogenic. |