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Article

Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patients

Intrathecal (IT) enzyme replacement therapy with recombinant human α-L-iduronidase (rhIDU) has been studied to treat glycosaminoglycan storage in the central nervous system of mucopolysaccharidosis (MPS) I dogs a...

Moin Vera, Steven Le, Shih-hsin Kan, Hermes Garban, David Naylor… in Pediatric Research (2013)