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Autologous CD34+ hematopoietic stem cells transduced with an hCD11b lentiviral vector encoding for the human N-sulfoglucosamine sulfohydrolase (SGSH) gene |
|---|---|
| Trade Name | |
| Orphan Indication | Mucopolysaccharidosis IIIA |
| USA Market Approval | USA |
| USA Designation Date | 2017-05-03 00:00:00 |
| Sponsor | Orchard Therapeutics North America;1118 Chess Drive, Suite A;Foster City, California, 94404 |
