Safety and efficacy of erythrocyte encapsulated thymidine phosphorylase in mitochondrial neurogastrointestinal encephalomyopathy

Safety and efficacy of erythrocyte encapsulated thymidine phosphorylase in mitochondrial neurogastrointestinal encephalomyopathy

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an ultra-rare autosomal recessive disorder of nucleoside metabolism that is caused by mutations in the nuclear thymidine phosphorylase gene (TYMP) gene, encoding for the enzyme thymidine phosphorylase. There are currently no approved t...

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Bibliografski detalji
Glavni autori: Levene, M, Bain, MD, Moran, NF, Nirmalananthan, N, Poulton, J, Scarpelli, M, Filosto, M, Mandel, H, Mackinnon, AD, Fairbanks, L, Pacitti, D, Bax, BE
Format: Journal article
Jezik:English
Izdano: MDPI 2019

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