Metabolomic profiling in treated mucopolysaccharidosis IH reveals candidate biomarkers and adjunctive therapeutic pathways
Troy C. Lund, Ryan H. Peretz, Patricia Dickson, Jennifer K. Yee, Michelina Iacovino, Kent D. Taylor, David A. Elashoff, Ellen B. Fung, Bradley Scott Miller, Paul J. Orchard, Lynda E. Polgreen
This study identifies metabolic differences between MPS IH and MPS IA patients treated with different therapies, highlighting potential biomarkers and therapeutic pathways.
medRxiv · Sep 7, 2026 · PreprintGenetic & Genomic Medicine