Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I
ED Kakkis, MF McEntee, A Schmidtchen… - … and molecular medicine, 1996 - Elsevier
Enzyme replacement is a potential therapy for mucopolysaccharidosis I (MPS I), a lysosomal
storage disorder caused by α-L-iduronidase deficiency. Previous work showed improvement
in the tissues of MPS I dogs treated intravenously for 3 months with recombinant human α-L-
iduronidase (25,000 units or∼ 0.1 mg/kg/week). We have now treated an MPS I-affected
dog for 13 months to assess the clinical effects of enzyme replacement. The treated dog
gained more weight, was more active, and had less joint stiffness than the untreated …
storage disorder caused by α-L-iduronidase deficiency. Previous work showed improvement
in the tissues of MPS I dogs treated intravenously for 3 months with recombinant human α-L-
iduronidase (25,000 units or∼ 0.1 mg/kg/week). We have now treated an MPS I-affected
dog for 13 months to assess the clinical effects of enzyme replacement. The treated dog
gained more weight, was more active, and had less joint stiffness than the untreated …
