Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry
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DOI: 10.1186/s13023-017-0635-z
Abstract: Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is a rare lysosomal storage disease with progressive multisystem manifestations caused by deficient activity of the enzyme iduronate-2-sulfatase. Disease-specific treatment is available in the form of… read more here.
Keywords: disease; hunter outcome; hos; survey hos ... See more keywords