Erythrocyte Encapsulated Thymidine Phosphorylase for the Treatment of Patients with Mitochondrial Neurogastrointestinal Encephalomyopathy: Study Protocol for a Multi-Centre, Multiple Dose, Open Label Trial

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an autosomal recessive disorder which primarily affects the gastrointestinal and nervous systems. This disease is caused by mutations in the nuclear TYMP gene, which encodes for thymidine phosphorylase, an enzyme required for the norma...

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Udgivet i:J Clin Med
Main Authors: Bax, Bridget E., Levene, Michelle, Bain, Murray D., Fairbanks, Lynette D., Filosto, Massimiliano, Kalkan Uçar, Sema, Klopstock, Thomas, Kornblum, Cornelia, Mandel, Hanna, Rahman, Shamima, Roubertie, Agathe, Scarpelli, Mauro, Sedgwick, Philip M., Baru, Moshe, Sellos-Moura, Marcia, Price, Jeanie, Horn, Patrick, Nirmalananthan, Niranjanan
Format: Artigo
Sprog:Inglês
Udgivet: MDPI 2019
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6722784/
https://ncbi.nlm.nih.gov/pubmed/31344955
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/jcm8081096
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