Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I

Article ID	Journal	Published Year	Pages	File Type
10833585	Molecular Genetics and Metabolism	2014	30 Pages	PDF

Abstract

The MPS I canine IA-rhIDUA results suggest that clinical studies should be performed to determine if IA-rhIDUA is a viable approach to ameliorating refractory orthopedic disease in human MPS I.

Keywords

Lysosomal storage disorder Orthopedic Therapy Canine Model Mucopolysaccharidosis

Related Topics

Life Sciences Biochemistry, Genetics and Molecular Biology Biochemistry

Preview

Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I

Authors

Raymond Y. Wang, Afshin Aminian, Michael F. McEntee, Shih-Hsin Kan, Calogera M. Simonaro, William C. Lamanna, Roger Lawrence, N. Matthew Ellinwood, Catalina Guerra, Steven Q. Le, Patricia I. Dickson, Jeffrey D. Esko,