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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Publicado en:J Clin Med
Autores principales: 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
Formato: Artigo
Lenguaje:Inglês
Publicado: MDPI 2019
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Article
Acceso en línea: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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