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...
Main Authors: | , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Wiley
2021-03-01
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Series: | JIMD Reports |
Subjects: | |
Online Access: | https://doi.org/10.1002/jmd2.12190 |