Total submissions: 3
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Undiagnosed Diseases Network, |
RCV000626052 | SCV000746672 | pathogenic | Charcot-Marie-Tooth disease axonal type 2S | 2017-03-24 | criteria provided, single submitter | research | This individual has been reported in PMID: 31020813. |
Gene |
RCV002225695 | SCV002504555 | pathogenic | not provided | 2019-10-17 | criteria provided, single submitter | clinical testing | Non-canonical splice site variant demonstrated to result in loss-of-function (Cassini et al., 2019); Observed in a patient in published literature with Charcot-Marie-Tooth phenotype with early onset symptoms (Cassini et al., 2019); This variant is associated with the following publications: (PMID: 31020813, 32190976) |
Labcorp Genetics |
RCV003767835 | SCV004578529 | likely pathogenic | Autosomal recessive distal spinal muscular atrophy 1; Charcot-Marie-Tooth disease axonal type 2S | 2023-07-09 | criteria provided, single submitter | clinical testing | Studies have shown that this variant results in activation of a cryptic splice site and introduces a premature termination codon (PMID: 31020813). The resulting mRNA is expected to undergo nonsense-mediated decay. In summary, the currently available evidence indicates that the variant is pathogenic, but additional data are needed to prove that conclusively. Therefore, this variant has been classified as Likely Pathogenic. ClinVar contains an entry for this variant (Variation ID: 522869). This sequence change falls in intron 8 of the IGHMBP2 gene. It does not directly change the encoded amino acid sequence of the IGHMBP2 protein. RNA analysis indicates that this variant induces altered splicing and may result in an absent or disrupted protein product. This variant is not present in population databases (gnomAD no frequency). This variant has been observed in individual(s) with clinical features of Charcot-Marie-Tooth disease (PMID: 31020813, 32190976). In at least one individual the data is consistent with being in trans (on the opposite chromosome) from a pathogenic variant. |