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 identify surrogate biomarkers of future change in orthopedic disease.

Methods

As part of a 9-year observational study of MPS I, range-of-motion (ROM), height, pelvic radiographs were measured annually. Biomarkers in year 1 were compared to healthy controls. Linear regression tested for associations of change in biomarkers over the first year with change in long-term outcomes.

Results

MPS I participants (N = 19) were age 5 to 16 years and on average 6.9 ± 2.9 years post treatment initiatio

SUBMITTER: Lund TC 

PROVIDER: S-EPMC7932872 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature