Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV002509878 | SCV002818521 | pathogenic | Phenylketonuria | 2022-12-09 | reviewed by expert panel | curation | This c.510-1G>C variant in PAH was detected in a patient with PKU with the pathogenic variant p.Arg111Ter (PMID: 28982351). This variant was absent in population databases. This is a canonical variant in the -1 splice acceptor of intron 5. Exon skipping or use of a cryptic splice site would disrupt reading frame with nonsense mediated decay predicted. In summary, this variant meets criteria to be classified as pathogenic for PAH. PAH-specific ACMG/AMP criteria applied: PVS1, PM2, PM3, PP4. |