Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV001789762 | SCV002032221 | likely pathogenic | Phenylketonuria | 2020-06-11 | reviewed by expert panel | curation | The c.832A>G (p.Thr278Ala) variant in PAH has been reported in multiple individuals with PKU and MHP (BH4 deficiency excluded). (PMID: 26503515, 30050108). This variant is absent in population databases. This variant was detected in the homozygous state in 1 patient (confirmed by parental testing). Computational evidence supports a deleterious effect. Another variant at this same amino acid is interpreted as pathogenic (p.Thr278Ile). In summary, this variant meets criteria to be classified as likely pathogenic for PAH. PAH-specific ACMG/AMP criteria applied: PP4_Moderate, PM2, PM5, PM3_supporting PP3. |
De |
RCV000089122 | SCV000119730 | not provided | not provided | no assertion provided | not provided |