Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV002260504 | SCV002540170 | pathogenic | Phenylketonuria | 2020-11-09 | reviewed by expert panel | curation | The c.1244A>T (p.Asp415Val) variant in PAH has been reported in 2 individuals with PKU, detected in trans with pathogenic variants c.1066-11G>A and c.47_48delCT (PMID: 21890392). This variant is absent in population databases. Computational prediction tools and conservation analysis support a deleterious effect. Another missense variant at the same amino acid is interpreted as pathogenic (p.Asp415Asn) In summary, this variant meets criteria to be classified as pathogenic for PAH. PAH-specific ACMG/AMP criteria applied: PM3_strong, PM2, PM5, PP3, PP4. |