Total submissions: 4
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Rady Children's Institute for Genomic Medicine, |
RCV000032991 | SCV000996226 | pathogenic | PGM1-congenital disorder of glycosylation | 2018-12-13 | criteria provided, single submitter | clinical testing | This nonsense variant found in exon 10 of 11 is predicted to result in loss of normal protein function. This variant has been previously reported as a homozygous change in two patients with Pierre Robin sequence, cleft palate, tachycardia, dilated cardiomyopathy, elevated creatine kinase, and elevated liver enzymes (PMID: 22492991, 24499211) and as a compound heterozygous change in trans with c.1172G>T p.Gly391Val in one patient with hypoglycemia, cleft palate, micrognathia, delayed speech development, elevated liver enzymes, and mild enlargment of the left ventricle (PMID: 29858906). Functional characterization of fibroblasts from both patients with a homozygous change demonstrated phosphoglucomutase activity of 7-8% as compared to controls (PMID: 22492991). It is present in the heterozygous state in the gnomAD population database at a frequency of 0.0012% (3/251206) and thus is presumed to be rare. Based on the available evidence, the c.1561C>T p.Arg521Ter variant is classified as pathogenic. |
Labcorp Genetics |
RCV000032991 | SCV001584124 | pathogenic | PGM1-congenital disorder of glycosylation | 2020-12-24 | criteria provided, single submitter | clinical testing | For these reasons, this variant has been classified as Pathogenic. Loss-of-function variants in PGM1 are known to be pathogenic (PMID: 22492991). This variant has been observed in individual(s) with PGM1-congenital disorder of glycosylation (PMID: 22492991, 24499211). ClinVar contains an entry for this variant (Variation ID: 39772). This variant is not present in population databases (ExAC no frequency). This sequence change creates a premature translational stop signal (p.Arg503*) in the PGM1 gene. It is expected to result in an absent or disrupted protein product. |
Gene |
RCV004719669 | SCV005325045 | pathogenic | not provided | 2024-02-21 | criteria provided, single submitter | clinical testing | Nonsense variant predicted to result in protein truncation or nonsense mediated decay in a gene for which loss of function is a known mechanism of disease; Not observed at significant frequency in large population cohorts (gnomAD); This variant is associated with the following publications: (PMID: 36007526, 24499211, 22492991, 29858906) |
OMIM | RCV000032991 | SCV000056770 | pathogenic | PGM1-congenital disorder of glycosylation | 2012-10-01 | no assertion criteria provided | literature only |