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Genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimized shRNA-insensitive wildtype PABPN1 |
|---|---|
| Trade Name | |
| Orphan Indication | Oculopharyngeal muscular dystrophy |
| EU Market Approval | EU |
| EU Designation Date | 2017-01-12 00:00:00 |
| Sponsor | Clinipace GmbH |
