recombinant adeno-associated virus vector serotype 9 expressing human N-Sulfoglucosamine Sulfohydrolase | |
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Trade Name | |
Orphan Indication | Sanfilippo Syndrome |
USA Market Approval | USA |
USA Designation Date | 2016-09-01 00:00:00 |
Sponsor | Research Institute at Nationwide Children's Hospital;Center for Gene Therapy, 700 Childrens Drive;Columbus, Ohio, 43205 |