Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV000106351 | SCV001146718 | pathogenic | Phenylketonuria | 2019-05-26 | reviewed by expert panel | curation | The c.196G>T (p.Glu66Ter) variant in PAH has been previously reported in one proband with classic PKU (PMID: 26666653) (PP4). The proband was heterozygous for the variant and also harbor the pathogenic allele (per ClinGen VCEP) c. 1315+1G>A; however, the phase of the variants was not confirmed via parental testing. (PM3_supporting). The sequence change results in a nonsense variant which occurs in exon 3 of 13 in the in the canonical transcript of PAH, a gene fulfilling the most recent criteria for LOF being a known disease mechanism (see PMID: 30192042) (PVS1). It is absent from control databases (PM2). In summary, this variant meets criteria to be classified as pathogenic for PAH. PAH-specific ACMG/AMP criteria applied: PVS1, PM2, PP4, PM3_supporting. |
Inserm U 954, |
RCV000106351 | SCV000143850 | probable-pathogenic | Phenylketonuria | no assertion criteria provided | not provided | Converted during submission to Likely pathogenic. |