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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...
Autores principales: | , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley & Sons, Inc.
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7932872/ https://www.ncbi.nlm.nih.gov/pubmed/33728251 http://dx.doi.org/10.1002/jmd2.12190 |