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Adenoassociated virus vector (AAV) carrying a modified AAV serotype 2 backbone and coding sequence of human thymidine phosphorylase preceded by a human thyroxin-binding globulin promoter |
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| Trade Name | |
| Orphan Indication | For the treatment of Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE) |
| USA Market Approval | USA |
| USA Designation Date | 2014-09-04 00:00:00 |
| Sponsor | Columbia University Medical Center;Department of Neurology, 630 West 168th Street, P&S; 4-423;New York, New York, 10032 |
