Pentosan polysulfate: a novel therapy for the mucopolysaccharidoses.

ABSTRACT:

Background

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.

Methodology/principal findings

Treatment began during prenatal development and at 1 and 6 months of age. All animals were treated until they were 9 months old. Significant reductions in the serum and tissue levels of several inflammatory markers (e.g., TNF-alph

SUBMITTER: Schuchman EH 

PROVIDER: S-EPMC3554761 | biostudies-literature | 2013

REPOSITORIES: biostudies-literature