Biomarkers for prediction of skeletal disease progression in mucopolysaccharidosis type I

Abstract Background Orthopedic disease progresses in mucopolysaccharidosis type I (MPS I), even with approved therapies and remains a major factor in persistent suffering and disability. Novel therapies and accurate predictors of response are needed. The primary objective of this study was to identi...

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Bibliographic Details
Main Authors: Troy C. Lund, Terence M. Doherty, Julie B. Eisengart, Rebecca L. Freese, Kyle D. Rudser, Ellen B. Fung, Bradley S. Miller, Klane K. White, Paul J. Orchard, Chester B. Whitley, Lynda E. Polgreen
Format: Article
Language:English
Published: Wiley 2021-03-01
Series:JIMD Reports
Subjects:
Online Access:https://doi.org/10.1002/jmd2.12190