Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey
Overall, adult patients with neuronopathic and non-neuronopathic MPS II had a high disease burden and requirement for surgeries, emphasizing the need to continue multidisciplinary management and regular assessments in adulthood. Further research into the differences in care needs of adult patients with MPS II is warranted. Trial registration NCT03292887 .
Authors
- Paul Harmatz (ORCID: https://orcid.org/0000-0001-5603-2085)
- Maurizio Scarpa (ORCID: https://orcid.org/0000-0002-0382-3251)
- Jennifer Audi
- Uma Ramaswami (ORCID: https://orcid.org/0000-0002-4703-7447)
- David Molter
- Shuan‐Pei Lin (ORCID: https://orcid.org/0000-0003-4023-5405)
- Bianca Link (ORCID: https://orcid.org/0000-0002-0217-5571)
- Jaco Botha (ORCID: https://orcid.org/0000-0002-8889-9959)
- Joseph Muenzer (ORCID: https://orcid.org/0000-0002-4035-6592)
- Barbara K. Burton
- Roberto Giugliani
- Hernán Amartino
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)
- Royal Free London NHS Foundation Trust (GB)
- Washington University in St. Louis (US)
- Mackay Memorial Hospital (TW)
- Takeda (Switzerland) (CH)
- Hospital Universitario Austral (AR)
- National Institute on Population Medical Genetics (BR)
- University Children's Hospital Zurich (CH)
- UCSF Benioff Children's Hospital (US)
- Takeda (Japan) (JP)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-07-01
- DOI
- https://doi.org/10.1186/s13023-024-03464-8
- Citations
- 1
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 1.16
Funders
- Takeda Pharmaceuticals International