Clinical characteristics and somatic burden of patients with mucopolysaccharidosis II with or without neurological involvement: An analysis from the Hunter Outcome Survey

Clinical characteristics and somatic burden of patients with mucopolysaccharidosis II with or without neurological involvement: An analysis from the Hunter Outcome Survey

Approximately two-thirds of patients with mucopolysaccharidosis II (MPS II; Hunter syndrome) have neuronopathic disease, with central nervous system involvement; one-third have non-neuronopathic disease. This analysis of data from the Hunter Outcome Survey (HOS) compared the clinical manifestations...

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Bibliographic Details
Main Authors: Heather Lau, Paul Harmatz, Jaco Botha, Jennifer Audi, Bianca Link
Format: Article
Language:English
Published: Elsevier 2023-12-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Mucopolysaccharidosis type II
Hunter Outcome Survey
Enzyme replacement therapy
Surgery
Neuronopathic phenotype
Non-neuronopathic phenotype
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426923000514

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http://www.sciencedirect.com/science/article/pii/S2214426923000514

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