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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.
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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  Data: Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey.
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  Data: <searchLink fieldCode="AU" term="%22Muenzer+J%22">Muenzer J</searchLink>; University of North Carolina at Chapel Hill, Chapel Hill, NC, 27514, USA. muenzer@med.unc.edu.<br /><searchLink fieldCode="AU" term="%22Amartino+H%22">Amartino H</searchLink>; Hospital Universitario Austral, Buenos Aires, Argentina.<br /><searchLink fieldCode="AU" term="%22Giugliani+R%22">Giugliani R</searchLink>; Department of Genetics UFRGS, Medical Genetics Service HCPA, INAGEMP, DASA Genomica and Casa Dos Raros, Porto Alegre, Brazil.<br /><searchLink fieldCode="AU" term="%22Harmatz+P%22">Harmatz P</searchLink>; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA.<br /><searchLink fieldCode="AU" term="%22Lin+SP%22">Lin SP</searchLink>; Mackay Memorial Hospital, Taipei, Taiwan.<br /><searchLink fieldCode="AU" term="%22Link+B%22">Link B</searchLink>; University Children's Hospital, Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Molter+D%22">Molter D</searchLink>; Washington University School of Medicine, St. Louis, MO, USA.<br /><searchLink fieldCode="AU" term="%22Ramaswami+U%22">Ramaswami U</searchLink>; Lysosomal Storage Disorders Unit, Royal Free London NHS Foundation Trust, London, UK.<br /><searchLink fieldCode="AU" term="%22Scarpa+M%22">Scarpa M</searchLink>; Udine University Hospital, Udine, Italy.<br /><searchLink fieldCode="AU" term="%22Botha+J%22">Botha J</searchLink>; Takeda Pharmaceuticals International AG, Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Audi+J%22">Audi J</searchLink>; Takeda Pharmaceuticals International AG, Zurich, Switzerland.; Takeda Pharmaceuticals International AG, Ultragenyx Europe GmbH, Lichstrasse, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Burton+BK%22">Burton BK</searchLink>; Ann & Robert H Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL, USA.
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  Data: <searchLink fieldCode="JN" term="%22101266602%22">Orphanet journal of rare diseases</searchLink> [Orphanet J Rare Dis] 2025 Jul 01; Vol. 20 (1), pp. 319. <i>Date of Electronic Publication: </i>2025 Jul 01.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22BioMed+Central%22">BioMed Central </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>101266602 <i>Publication Model: </i>Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1750-1172 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2217501172%22">17501172 </searchLink><i>NLM ISO Abbreviation: </i>Orphanet J Rare Dis <i>Subsets: </i>MEDLINE
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              Text: 2025 Jul 01
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