Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
University Children's Hospital, |
RCV000236522 | SCV000262801 | pathogenic | Methylmalonic aciduria due to methylmalonyl-CoA mutase deficiency | criteria provided, single submitter | clinical testing | ||
Women's Health and Genetics/Laboratory Corporation of America, |
RCV005406950 | SCV006074412 | likely pathogenic | Methylmalonic acidemia | 2025-04-23 | criteria provided, single submitter | clinical testing | Variant summary: MMUT c.1164T>A (p.Asn388Lys) results in a non-conservative amino acid change in the encoded protein sequence. Five of five in-silico tools predict a damaging effect of the variant on protein function. The variant was absent in 251242 control chromosomes. c.1164T>A has been observed in at least one homozygous individual affected with Methylmalonic Acidemia (e.g. Forny_2016). These data indicate that the variant may be associated with disease. At least one publication reports experimental evidence evaluating an impact on protein function. The most pronounced variant effect results in <10% of normal enzyme activity (e.g. Forny_2016). The following publication has been ascertained in the context of this evaluation (PMID: 27167370). ClinVar contains an entry for this variant (Variation ID: 222929). Based on the evidence outlined above, the variant was classified as likely pathogenic. |