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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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Bibliografske podrobnosti
izdano v:J Clin Med
Main Authors: Levene, Michelle, Bain, Murray D., Moran, Nicholas F., Nirmalananthan, Niranjanan, Poulton, Joanna, Scarpelli, Mauro, Filosto, Massimiliano, Mandel, Hanna, MacKinnon, Andrew D., Fairbanks, Lynette, Pacitti, Dario, Bax, Bridget E
Format: Artigo
Jezik:Inglês
Izdano: MDPI 2019
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC6517976/
https://ncbi.nlm.nih.gov/pubmed/30959750
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/jcm8040457
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