Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I.

@article{Kakkis1996LongtermAH,
  title={Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I.},
  author={E. Kakkis and Michael F. McEntee and Artur Schmidtchen and Elizabeth F. Neufeld and D. A. Ward and Rebecca E Gompf and Stephen Kania and C Bedolla and S. H. Chien and Robert M Shull},
  journal={Biochemical and molecular medicine},
  year={1996},
  volume={58 2},
  pages={156-67}
}
Enzyme replacement is a potential therapy for mucopolysaccharidosis I (MPS I), a lysosomal storage disorder caused by alpha-L-iduronidase deficiency. Previous work showed improvement in the tissues of MPS I dogs treated intravenously for 3 months with recombinant human alpha-L-iduronidase (25,000 units or approximately 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… CONTINUE READING
53 Citations
0 References
Similar Papers

Citations

Publications citing this paper.
Showing 1-10 of 53 extracted citations

Similar Papers

Loading similar papers…