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Adeno-associated virus serotype 9 vector containing human Iduronate-2-sulfatase transgene

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Adeno-associated virus serotype 9 vector containing human Iduronate-2-sulfatase transgene is designated as an orphan drug by the US FDA on 7/16/2015, used in treatment of mucopolysaccharidosis type II (Hunter syndrome), manufactured / distributed by Esteve Pharmaceuticals, S.A,


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