Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV001543636 | SCV001762304 | pathogenic | Phenylketonuria | 2020-07-02 | reviewed by expert panel | curation | The c.202A>G (p.Arg68Gly) variant in PAH has been reported in multiple individuals with PKU (BH4 deficiency excluded). (PMID: 21147011). This variant is absent in population databases. This variant was detected with multiple pathogenic variants: R243Q (PMID: 10495930); p.R408W (3 patients including 2 siblings); c.842+1G>A (2 patients) PMID: 24350308. Computational prediction tools and conservation analysis support a deleterious effect on the protein. Another missense variant at the same amino acid (p.Arg68Ser) is pathogenic. In summary, this variant meets criteria to be classified as pathogenic for PAH. PAH-specific ACMG/AMP criteria applied: PM3_strong, PM2, PM5, PP4_Moderate, PP3, PP1. |
De |
RCV000088870 | SCV000119466 | not provided | not provided | no assertion provided | not provided |