Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Vockley Lab, |
RCV002264901 | SCV002546245 | pathogenic | Mitochondrial trifunctional protein deficiency | 2022-04-20 | criteria provided, single submitter | research | The point deletion with frameshift variant c.693delC in exon 9 (p.A232Lfs*20) has been initially described by Spiekerkoetter et al. (2003) in the same patient as the present submission. It was found in a compound heterozygous state with the missense variant c.881C>G (p.P294R) in exon 10. They described the patient as a male Caucasian patient presenting the hepatic form of the disease, who was 5-month old at the onset and 1-year-5-month old when the paper was published – in the latter occasion he was asymptomatic. In the patient description, the authors refer hypoglycemia, but not cardiomyopathy, skeletal myopathy, neuropathy or maternal HELLP syndrome. |