Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry.

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Term Occurence Count Dictionary
lysosomal storage disease 2 endocrinologydiseases
mucopolysaccharidosis 3 endocrinologydiseases
Fabry disease 1 endocrinologydiseases

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Fabry disease 3356 clinical course. Patient registries have been established for a number of LSD, including Gaucher disease, Fabry disease , Pompe disease, and mucopolysaccharidosis types I, II, IV, and VI [[5]–[12]], and have provided important
lysosomal storage disease 598 (collection): /2017AbstractMucopolysaccharidosis 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.
lysosomal storage disease 3084 [[3], [4]].One group of diseases for which patient registries have been particularly valuable is the lysosomal storage disease s (LSD). These are rare, inherited, progressive disorders caused by defects in lysosomal function, and
mucopolysaccharidosis 3390 been established for a number of LSD, including Gaucher disease, Fabry disease, Pompe disease, and mucopolysaccharidosis types I, II, IV, and VI [[5]–[12]], and have provided important insights into the natural history
mucopolysaccharidosis 4383 2007, and stabilizes many of the somatic signs and symptoms of MPS II [[18]–[22]].Table 1What is mucopolysaccharidosis type II (MPS II)?• First described in two brothers by Dr Charles A. Hunter in 1917.• Caused by deficient
mucopolysaccharidosis 9547 questionnaireHS-FOCUS Hunter Syndrome Functional Outcomes for Clinical Understanding Scale, MPS II mucopolysaccharidosis type IIHOS is operational in many countries around the world. The registry has grown significantly over

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