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Published in 2019 at "Molecular Genetics and Metabolism"
DOI: 10.1016/j.ymgme.2018.12.210
Abstract: Hunter syndrome is a rare X-linked lysosomal disorder associated with progressive multisystem involvement. Ages of onset vary, as do disease course and phenotypic severity. The early progressive phenotype involves delayed developmental milestones with worsening intellectual…
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Keywords:
early progressive;
progressive mps;
feasibility quantifying;
behavior early ... See more keywords