Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV000668178 | SCV001443301 | uncertain significance | Glycogen storage disease, type II | 2023-04-07 | reviewed by expert panel | curation | The NM_000152.5:c.2853G>A (p.Trp951Ter) variant in GAA is a nonsense variant predicted to cause a premature stop codon in the penultimate codon of the gene, thereby escaping nonsense-mediated decay and resulting in a 2 amino acid C-terminal truncation of GAA (PVS1_Moderate). The variant is absent in gnomAD v2.1.1 (PM2_Supporting). To our knowledge, this variant has not been reported in the literature in a patient with Pompe disease. There is a ClinVar entry for this variant (Variation ID: 552839). In summary, this variant meets the criteria to be classified as a variant of uncertain significance for Pompe disease. GAA-specific ACMG/AMP criteria met, based on the specifications of the ClinGen Lysosomal Diseases VCEP: PVS1_Moderate, PM2_Supporting. (Classification approved by the ClinGen Lysosomal Diseases VCEP on April 7, 2023). |
Counsyl | RCV000668178 | SCV000792736 | uncertain significance | Glycogen storage disease, type II | 2017-07-11 | criteria provided, single submitter | clinical testing |