Sep 2026
Metabolomic profiling in treated mucopolysaccharidosis IH reveals candidate biomarkers and adjunctive therapeutic pathways
Persistent metabolic differences were observed in MPS IH treated with HCT compared to MPS IA treated with ERT, pointing towards additional biological processes that may contribute to disease progression in MPS IH after HCT.
Troy C. Lund, Ryan H. Peretz, Patricia I. Dickson et al.
· medRxiv · 0 citations