Orphan designation: Adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase-alpha gene Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome), 24/01/2013 Withdrawn
Orphan designation: Adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase-alpha gene Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome), 24/01/2013 Withdrawn
