Treatment of mucopolysaccharidosis I with fully-human glycosylated human alpha-l-iduronidase (IDUA)

摘要

Compositions and methods are described for the delivery of a fully human-glycosylated (HuGly) α-L-iduronidase (IDUA) to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis I (MPS I).