Biomarkers for prediction of skeletal disease progression in mucopolysaccharidosis type I

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 identify surro...

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Bibliografiske detaljer
Udgivet i:JIMD Rep
Main Authors: Lund, Troy C., Doherty, Terence M., Eisengart, Julie B., Freese, Rebecca L., Rudser, Kyle D., Fung, Ellen B., Miller, Bradley S., White, Klane K., Orchard, Paul J., Whitley, Chester B., Polgreen, Lynda E.
Format: Artigo
Sprog:Inglês
Udgivet: John Wiley & Sons, Inc. 2020
Fag:
Research Reports
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7932872/
https://ncbi.nlm.nih.gov/pubmed/33728251
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jmd2.12190
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