Journal article
Safety and efficacy of erythrocyte encapsulated thymidine phosphorylase in mitochondrial neurogastrointestinal encephalomyopathy
- Abstract:
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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 treatments for MNGIE. The aim of this study was to investigate the safety, tolerability, and efficacy of an enzyme replacement therapy for the treatment of MNGIE. In this single centre study, three ...
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- Publication status:
- Published
- Peer review status:
- Peer reviewed
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(Version of record, pdf, 9.7MB)
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- Publisher copy:
- 10.3390/jcm8040457
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Authors
Funding
Bibliographic Details
- Publisher:
- MDPI Publisher's website
- Journal:
- Journal of Clinical Medicine Journal website
- Volume:
- 8
- Issue:
- 4
- Article number:
- 457
- Publication date:
- 2019-04-05
- Acceptance date:
- 2019-04-03
- DOI:
- EISSN:
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2077-0383
- Pmid:
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30959750
- Source identifiers:
-
992425
Item Description
- Language:
- English
- Keywords:
- Pubs id:
-
pubs:992425
- UUID:
-
uuid:a725ef35-9563-41a5-a018-b5025e56a4e7
- Local pid:
- pubs:992425
- Deposit date:
- 2019-06-25
Terms of use
- Copyright holder:
- Levene et al
- Copyright date:
- 2019
- Notes:
- © 2019 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
- Licence:
- CC Attribution (CC BY)
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