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Community register of orphan medicinal products


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Self-complementary adeno-associated viral vector serotype 9 containing the SGSH gene

EU orphan designation number: EU/3/16/1761   
Active ingredient: Self-complementary adeno-associated viral vector serotype 9 containing the SGSH gene
Indication: Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome)
Sponsor: Ser-mes Planificación SL
Rufino González 14, Esc.1ª-2ºD, 28037 Madrid, 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
18/10/2016 Orphan designation EMA/OD/164/16 (2016)6753 of 14/10/2016