Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey

Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey

Abstract Background Mucopolysaccharidosis II (MPS II) is a rare, life-limiting lysosomal storage disease caused by deficient iduronate-2-sulfatase activity. The current standard of care for MPS II is intravenous enzyme replacement therapy (ERT), which has been shown to improve somatic signs and symp...

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Bibliographic Details
Main Authors: Joseph Muenzer, Hernan Amartino, Roberto Giugliani, Paul Harmatz, Shuan-Pei Lin, Bianca Link, David Molter, Uma Ramaswami, Maurizio Scarpa, Jaco Botha, Jennifer Audi, Barbara K. Burton
Format: Article
Language:English
Published: BMC 2025-07-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Adults
Cognitive Impairment
Hunter Syndrome
Mucopolysaccharidosis type II
Neuronopathic
Non-neuronopathic
Online Access:https://doi.org/10.1186/s13023-024-03464-8
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