%0 Journal Article
%T Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes
%J -
%D 2019
%R https://doi.org/10.1038/s41436-019-0522-1
%X Abnormalities in cerebrospinal fluid (CSF) have been reported in Hurler syndrome, a fatal neurodegenerative lysosomal disorder. While no biomarker has predicted neurocognitive response to treatment, one of these abnormalities, glycosaminoglycan nonreducing ends (NREs), holds promise to monitor therapeutic efficacy. A trial of intrathecal enzyme replacement therapy (ERT) added to standard treatment enabled tracking of CSF abnormalities, including NREs. We evaluated safety, biomarker response, and neurocognitive correlates of change
%U https://www.nature.com/articles/s41436-019-0522-1