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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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6517976/
https://www.ncbi.nlm.nih.gov/pubmed/30959750
http://dx.doi.org/10.3390/jcm8040457