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recombinant, adeno-associated virus serotype 5 vector, containing the transgene, which encodes for the human protein, frataxin |
|---|---|
| Trade Name | |
| Orphan Indication | Friedreich Ataxia |
| USA Market Approval | USA |
| USA Designation Date | 2016-07-25 00:00:00 |
| Sponsor | Agilis Biotherapeuties, LLC;245 First Street, Suite 1800;Cambridge, Massachusetts, 02142 |
