Clinical characteristics and real-world outcomes in patients with mucopolysaccharidosis II over 18 years: final report of the Hunter Outcome Survey
Data from HOS, collected for over 18 years, represent the largest dataset of patients with MPS II to date. The effectiveness and safety profile of idursulfase support its use for the long-term treatment of patients with MPS II.
Authors
- Paul Harmatz (ORCID: https://orcid.org/0000-0001-5603-2085)
- David Molter
- Shuan‐Pei Lin (ORCID: https://orcid.org/0000-0003-4023-5405)
- Anna Tylki‐Szymańska (ORCID: https://orcid.org/0000-0002-8935-1768)
- Bianca Link (ORCID: https://orcid.org/0000-0002-0217-5571)
- Jaco Botha (ORCID: https://orcid.org/0000-0001-8974-2438)
- David Whiteman (ORCID: https://orcid.org/0000-0001-6383-9338)
- Siddharth Jain (ORCID: https://orcid.org/0000-0001-6122-3775)
- Christoph Kampmann (ORCID: https://orcid.org/0000-0002-2929-2970)
- Joseph Muenzer (ORCID: https://orcid.org/0000-0002-4035-6592)
- Roberto Giugliani (ORCID: https://orcid.org/0000-0001-9655-3686)
- Maurizio Scarpa
- Barbara K. Burton
- Hernán Amartino
- Julian Raiman
Institutions
- Northwestern University (US)
- University of North Carolina at Chapel Hill (US)
- University of Udine (IT)
- Lurie Children's Hospital (US)
- Universidade Federal do Rio Grande do Sul (BR)
- Johannes Gutenberg University Mainz (DE)
- Washington University in St. Louis (US)
- Mackay Memorial Hospital (TW)
- Takeda (Switzerland) (CH)
- Hospital Universitario Austral (AR)
- Birmingham Children's Hospital (GB)
- Children's Memorial Health Institute (PL)
- National Institute on Population Medical Genetics (BR)
- University Children's Hospital Zurich (CH)
- UCSF Benioff Children's Hospital (US)
- Takeda (United States) (US)
Publication Details
- Journal
- Molecular Genetics and Metabolism
- Published
- 2025-11-03
- DOI
- https://doi.org/10.1016/j.ymgme.2025.109284
- Citations
- 1
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 1.16