Biomarkers for prediction of skeletal disease progression in mucopolysaccharidosis type I.
Troy C LundTerence M DohertyJulie B EisengartRebecca L FreeseKyle D RudserEllen B FungBradley S MillerKlane K WhitePaul J OrchardChester B WhitleyLynda E PolgreenPublished in: JIMD reports (2020)
Inflammatory cytokines are high in MPS I. IL-6 and PYD were associated with progression in joint contracture, short stature, and hip dysplasia over time. Once validated, these biomarkers may prove useful for predicting response to treatment of skeletal disease in MPS I.