Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV000106369 | SCV001146744 | pathogenic | Phenylketonuria | 2019-07-14 | reviewed by expert panel | curation | The c.837del frameshift variant has been identified in at least 2 probands with classic PKU, BH4 deficiency not excluded (PMIDs: 10598814, 26666653). It has been detected in trans with pathogenic variants V388M (PMID: 10598814) and c.1315+1G>A (PMID: 26666653). This variant is absent from 1000G, Exac, and gnomAD databases. C.837delC generates a frameshift predicted to result in a premature stop codon 61 residues downstream in exon 10 and undergo NMD. In summary, this variant meets criteria to be classified as pathogenic for PAH. PAH-specific ACMG/AMP criteria applied: PVS1, PM3, PM2, PP4. |
Inserm U 954, |
RCV000106369 | SCV000143869 | probable-pathogenic | Phenylketonuria | no assertion criteria provided | not provided | Converted during submission to Likely pathogenic. |