Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV001375885 | SCV001572841 | pathogenic | Phenylketonuria | 2020-11-24 | reviewed by expert panel | curation | This variant c.667_674dup (p.Gln226ThrfsTer?) in PAH was documented in a patient with PAH deficiency (>120 μmol/L Phe) in the Czech Republic (PMID: 23357515). This is a frameshift variant in exon 6 out of 13 coding exons, predicted to undergo nonsense mediated decay (NMD). The exon is present in biologically-relevant transcripts. This variant is absent in population databases. In summary, this variant meets criteria to be classified as pathogenic for PAH. PAH-specific ACMG/AMP criteria applied: PVS1, PM2, PP4 |