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Adeno associated viral vector serotype rh.10 carrying the human SGSH and SUMF1 cDNAs

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Adeno associated viral vector serotype rh.10 carrying the human SGSH and SUMF1 cDNAs is designated as an orphan drug by the US FDA on 5/6/2013, used in treatment of mucopolysaccharidosis type IIIA (Sanfilippo type A syndrome), manufactured / distributed by [[Lysogene


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