Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Labcorp Genetics |
RCV001376754 | SCV001573917 | pathogenic | Glycogen storage disease, type II | 2023-06-27 | criteria provided, single submitter | clinical testing | This variant has been observed in individual(s) with clinical features of Pompe disease (PMID: 7717400; Invitae). In at least one individual the data is consistent with being in trans (on the opposite chromosome) from a pathogenic variant. This variant is not present in population databases (gnomAD no frequency). This variant, c.2707_2709del, results in the deletion of 1 amino acid(s) of the GAA protein (p.Lys903del), but otherwise preserves the integrity of the reading frame. ClinVar contains an entry for this variant (Variation ID: 4028). For these reasons, this variant has been classified as Pathogenic. Experimental studies have shown that this variant affects GAA function (PMID: 7717400, 21972175). Algorithms developed to predict the effect of variants on protein structure and function are not available or were not evaluated for this variant. |
OMIM | RCV000004243 | SCV000024409 | pathogenic | Glycogen storage disease type II, infantile | 1995-04-01 | no assertion criteria provided | literature only |