Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV001269069 | SCV001448293 | uncertain significance | Phenylketonuria | 2020-10-15 | reviewed by expert panel | curation | This c.706+5G>A variant in PAH was reported in one patient with hyperphenylalaninemia. Tetrahydrobiopterin deficiency was ruled out with urinary pterin analysis and a DHPR activity assay (PMID: 29499199). This variant is absent from population databases. Multiple lines of computation evidence support a deleterious effect. In summary, this variant meets criteria to be classified as Uncertain Significance for PAH. PAH-specific ACMG/AMP criteria applied: PM2, PP4_moderate, and PP3. |