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Female Patients With Mucopolysaccharidosis II (MPS II): Insights From the Hunter Outcome Survey

ABSTRACT Mucopolysaccharidosis II is a rare, X‐linked disease, with very few reports of affected female patients. Natural history data describe a predominantly male population, and appropriate disease characterization in female patients is lacking. This analysis explores the somatic disease burden a...

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Principais autores: Barbara K. Burton, Hernan Amartino, Roberto Giugliani, Christoph Kampmann, Julian Raiman, Maurizio Scarpa, Anna Tylki‐Szymańska, Jennifer Audi, Jaco Botha, Siddarth Jain, Joseph Muenzer
Formato: Artigo
Idioma:Inglês
Publicado em: Wiley 2026-01-01
coleção:JIMD Reports
Assuntos:
enzyme replacement therapy
Hunter Outcome Survey
Hunter syndrome
idursulfase
mucopolysaccharidosis type II
X‐linked inheritance
Acesso em linha:https://doi.org/10.1002/jmd2.70046
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