Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV001093525 | SCV001250565 | uncertain significance | Phenylketonuria | 2019-11-22 | reviewed by expert panel | curation | This c.61-3T>C variant in PAH was reported twice in the Chinese PKU population, although this may be the same patient (PMID: 26503515; 28982351). DHPR activity, biopterin and/or pteridine analysis was performed to rule out other causes of hyperphenylalaninemia. This variant is absent from the population databases ExAC and gnomAD. However, in silico splicing predictions suggest no splicing impact (HSP, MaxENT, TraP). In summary, this variant meets criteria to be classified as uncertain significance for PAH. PAH-specific ACMG/AMP criteria applied: PP4_moderate, PM2, PM3, BP4. |