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Community register of orphan medicinal products


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Adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase alpha gene

EU orphan designation number: EU/3/12/1095   
Active ingredient: Adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase alpha gene
Indication: Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Sponsor: Laboratorios del Dr Esteve, S.A.
Av. Mare de Déu de Montserrat 221, 08041 Barcelona, España

   Public summary of scientific opinion    

European Commission proceduresGoto top of the page

Close date procedure Procedure type EMEA number Decision summary publ decision docs annex
28/01/2013 Orphan designation EMA/OD/150/12 (2013)484 of 24/01/2013