Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Molecular Genetics Department, |
RCV003986039 | SCV004801877 | likely pathogenic | Smith-Magenis syndrome | criteria provided, single submitter | clinical testing | A previously undescribed nucleotide variant creates a frameshift p.Ala123GlyfsTer20 in the RAI1 gene. The variant was observed in heterozygous state in an individual affected with polycystic kidney disease, hypertrophic cardiomyopathy, and dysmorphic features. Loss-of-function variants are reported in patients with Smith-Magenis syndrome, 182290. The variant is not present in population database (gnomAD no frequency). In summary, the currently available evidence indicates that the variant is pathogenic, but additional data are needed to prove that conclusively. Therefore, this variant has been classified as likely pathogenic. |