Open-label, single-center, clinical study evaluating the safety, tolerability and clinical effects of pentosan polysulfate sodium in subjects with mucopolysaccharidosis I
First published: 11 March 2024
https://onlinelibrary.wiley.com/doi/10.1002/jimd.12715
"Decreased GAG fragments and cartilage degradation biomarkers, and positive PROMIS outcomes support continued study of PPS as a potential disease-modifying treatment for MPS I with improved pain and function outcomes."
Open-label, single-center, clinical study evaluating the safety,...
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