Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV002509879 | SCV002818523 | likely pathogenic | Phenylketonuria | 2022-12-09 | reviewed by expert panel | curation | This variant c.1316-1G>C in PAH was detected in a patient with classic PKU with the pathogenic variant p.Arg243Gln (PMID: 28982351). This variant was absent in population databases. This is a canonical variant in the -1 splice acceptor of intron 12; this would alter a region that is critical to protein function with nonsense mediated decay not predicted. In summary, this variant meets criteria to be classified as likely pathogenic for PAH. PAH-specific ACMG/AMP criteria applied: PVS1_Strong, PM2, PM3, PP4. |