Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey.
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| Title: | Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey. |
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| Authors: | Muenzer J; University of North Carolina at Chapel Hill, Chapel Hill, NC, 27514, USA. muenzer@med.unc.edu., Amartino H; Hospital Universitario Austral, Buenos Aires, Argentina., Giugliani R; Department of Genetics UFRGS, Medical Genetics Service HCPA, INAGEMP, DASA Genomica and Casa Dos Raros, Porto Alegre, Brazil., Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA., Lin SP; Mackay Memorial Hospital, Taipei, Taiwan., Link B; University Children's Hospital, Zurich, Switzerland., Molter D; Washington University School of Medicine, St. Louis, MO, USA., Ramaswami U; Lysosomal Storage Disorders Unit, Royal Free London NHS Foundation Trust, London, UK., Scarpa M; Udine University Hospital, Udine, Italy., Botha J; Takeda Pharmaceuticals International AG, Zurich, Switzerland., Audi J; Takeda Pharmaceuticals International AG, Zurich, Switzerland.; Takeda Pharmaceuticals International AG, Ultragenyx Europe GmbH, Lichstrasse, Basel, Switzerland., Burton BK; Ann & Robert H Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL, USA. |
| Source: | Orphanet journal of rare diseases [Orphanet J Rare Dis] 2025 Jul 01; Vol. 20 (1), pp. 319. Date of Electronic Publication: 2025 Jul 01. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172 (Electronic) Linking ISSN: 17501172 NLM ISO Abbreviation: Orphanet J Rare Dis Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 1750-1172 |
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| DOI: | 10.1186/s13023-024-03464-8 |
