- Yang, Jamie;
- Shaybekyan, Hapet;
- Zhao, Yan;
- Kang, Xuedong;
- Fishbein, Gregory;
- Khanlou, Negar;
- Alejos, Juan;
- Halnon, Nancy;
- Satou, Gary;
- Biniwale, Reshma;
- Lee, Hane;
- Van Arsdell, Glen;
- Nelson, Stanley;
- Touma, Marlin
We report a case of hypertrophic cardiomyopathy and lactic acidosis in a 3-year-old female. Cardiac and skeletal muscles biopsies exhibited mitochondrial hyperplasia with decreased complex IV activity. Whole exome sequencing identified compound heterozygous variants, p.Arg333Trp and p.Val119Leu, in TSFM, a nuclear gene that encodes a mitochondrial translation elongation factor, resulting in impaired oxidative phosphorylation and juvenile hypertrophic cardiomyopathy.