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Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an autosomal-recessive, multisystem mitochondrial disease that leads to premature death. TYMP mutation causes loss of thymidine phosphorylase (TP) activity, which results in an increased level of deoxyuridine in plasma and tissues. As a result, there is depletion of mitochondrial DNA, which leads to progressive mitochondrial oxidative phosphorylation dysfunction with gastrointestinal dysmotility, cachexia, peripheral neuropathy, skeletal myopathy, ophthalmoparesis, and ptosis over the course of 2 to 4 decades. Researchers conducted a retrospective analysis of data in all MNGIE patients worldwide who underwent allogenic hematopoietic stem cell (HSCT) transplantation between 200…