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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Detalhes bibliográficos
Publicado no: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.
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley & Sons, Inc. 2020
Assuntos:
Research Reports
Acesso em linha: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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