Edward H. Schuchman, Yi Ge, Alon Lai, Yury Borisov, Meghan Faillace, Efrat Eliyahu, Xingxuan He, James Iatridis, Helen Vlassara, Gary Striker, Calogera M. Simonaro

Pentosan polysulfate (PPS) is an FDA-approved, oral medication with anti-inflammatory and pro-chondrogenic properties. We have previously shown that animal models of the mucopolysaccharidoses (MPS) exhibit significant inflammatory disease, contributing to cartilage degeneration. Enzyme replacement therapy (ERT) only partly reduced inflammation, and anti-TNF-alpha antibody therapy significantly enhanced clinical and pathological outcomes. Here we describe the use of PPS for the treatment of MPS type VI rats.