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non-replicating recombinant adeno-associated virus vector containing 4 antisense sequences designed to target exon 2 in the human DMD gene under the control of U7 small nuclear non-coding RNA |
|---|---|
| Trade Name | |
| Orphan Indication | Duchenne muscular dystrophy |
| USA Market Approval | USA |
| USA Designation Date | 2017-09-19 00:00:00 |
| Sponsor | Nationwide Children's Hospital;Drug and Device Development Services, 700 Children's Drive;Columbus, Ohio, 43205 |
