Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV001093521 | SCV001250561 | pathogenic | Phenylketonuria | 2019-10-18 | reviewed by expert panel | curation | This c.353-2A>G (IVS3-2A>G) variant in PAH was reported in two Chinese patients with PAH deficiency (PMID: 26503515). DHPR activity, biopterin and/or pteridine analysis was performed to rule out other causes of hyperphenylalaninemia. This variant is absent from population databases ExAC and gnomAD. This variant in the -2 splice acceptor site of IVS3 results in exon skipping or use of a cryptic splice site. In summary, this variant meets criteria to be classified as pathogenic for PAH. PAH-specific ACMG/AMP criteria applied: PP4_moderate, PM2, PVS1_very strong. |