Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
National Newborn Screening Laboratory, |
RCV001837053 | SCV002097376 | likely pathogenic | Maple syrup urine disease | criteria provided, single submitter | clinical testing | This variant is located within the ±2 bp from a splice site in the BCKDHA gene, where loss of function is a known disease mechanism. This variant is not present in population databases (gnomAD). It was found in a homozygous state in a patient with biochemical analysis supporting the diagnosis of MSUD (Pre-treatment plasma aminogram: Leucine: 1667umol/L, Valine: 522umol/L, Isoleucine: 454umol/L). |