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Genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimized shRNA-insensitive wildtype PABPN1

EU orphan designation number: EU/3/16/1816   
Active ingredient: Genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimized shRNA-insensitive wildtype PABPN1
Indication: Treatment of oculopharyngeal muscular dystrophy
Sponsor: Clinipace GmbH
Helfmann-Park 10, 65760 Eschborn, Deutschland

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Close date procedure Procedure type EMEA number Decision summary publ decision docs annex
16/01/2017 Orphan designation EMA/OD/194/16 (2017)220 of 12/01/2017