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Long-term outcomes of elosulfase alfa enzyme replacement therapy in adults with MPS IVA: a sub-analysis of the Morquio A Registry Study (MARS).

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Title: Long-term outcomes of elosulfase alfa enzyme replacement therapy in adults with MPS IVA: a sub-analysis of the Morquio A Registry Study (MARS).
Authors: Stepien KM; Northern Care Alliance NHS Foundation Trust, Salford, UK. karolina.stepien@nca.nhs.uk., Burton BK; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA., Bober MB; Nemours/Alfred I. DuPont Hospital for Children, Wilmington, DE, USA., Campeau PM; CHU Sainte-Justine Research Center, Montreal, Quebec, Canada., Ellaway C; Sydney Children's Hospital Network, The Children's Hospital at Westmead, Westmead, Sydney, NSW, Australia., Bhattacharya K; Sydney Children's Hospital Network, The Children's Hospital at Westmead, Westmead, Sydney, NSW, Australia., Guffon N; Reference Centre of Inherited Metabolic Disease, Hospices Civils de Lyon, Lyon, France., Hinds D; BioMarin Pharmaceutical Inc., Novato, CA, USA., Hunt A; BioMarin Pharmaceutical Inc., Novato, CA, USA., Lail A; BioMarin Pharmaceutical Inc., Novato, CA, USA., Lin SP; MacKay Children's Hospital, Taipei, Taiwan., Magner M; Department of Pediatrics and Inherited Metabolic Disorders, General University Hospital and First Faculty of Medicine, Charles University, Prague, Czech Republic., Murphy E; National Hospital for Neurology and Neurosurgery, London, UK., Reisewitz P; BioMarin Pharmaceutical Inc., Novato, CA, USA., Mitchell JJ; McGill University Health Centre, Montreal, Quebec, Canada.
Source: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2025 Oct 30; Vol. 20 (1), pp. 548. Date of Electronic Publication: 2025 Oct 30.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
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: Long-term outcomes of elosulfase alfa enzyme replacement therapy in adults with MPS IVA: a sub-analysis of the Morquio A Registry Study (MARS).
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  Data: <searchLink fieldCode="AU" term="%22Stepien+KM%22">Stepien KM</searchLink>; Northern Care Alliance NHS Foundation Trust, Salford, UK. karolina.stepien@nca.nhs.uk.<br /><searchLink fieldCode="AU" term="%22Burton+BK%22">Burton BK</searchLink>; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.<br /><searchLink fieldCode="AU" term="%22Bober+MB%22">Bober MB</searchLink>; Nemours/Alfred I. DuPont Hospital for Children, Wilmington, DE, USA.<br /><searchLink fieldCode="AU" term="%22Campeau+PM%22">Campeau PM</searchLink>; CHU Sainte-Justine Research Center, Montreal, Quebec, Canada.<br /><searchLink fieldCode="AU" term="%22Ellaway+C%22">Ellaway C</searchLink>; Sydney Children's Hospital Network, The Children's Hospital at Westmead, Westmead, Sydney, NSW, Australia.<br /><searchLink fieldCode="AU" term="%22Bhattacharya+K%22">Bhattacharya K</searchLink>; Sydney Children's Hospital Network, The Children's Hospital at Westmead, Westmead, Sydney, NSW, Australia.<br /><searchLink fieldCode="AU" term="%22Guffon+N%22">Guffon N</searchLink>; Reference Centre of Inherited Metabolic Disease, Hospices Civils de Lyon, Lyon, France.<br /><searchLink fieldCode="AU" term="%22Hinds+D%22">Hinds D</searchLink>; BioMarin Pharmaceutical Inc., Novato, CA, USA.<br /><searchLink fieldCode="AU" term="%22Hunt+A%22">Hunt A</searchLink>; BioMarin Pharmaceutical Inc., Novato, CA, USA.<br /><searchLink fieldCode="AU" term="%22Lail+A%22">Lail A</searchLink>; BioMarin Pharmaceutical Inc., Novato, CA, USA.<br /><searchLink fieldCode="AU" term="%22Lin+SP%22">Lin SP</searchLink>; MacKay Children's Hospital, Taipei, Taiwan.<br /><searchLink fieldCode="AU" term="%22Magner+M%22">Magner M</searchLink>; Department of Pediatrics and Inherited Metabolic Disorders, General University Hospital and First Faculty of Medicine, Charles University, Prague, Czech Republic.<br /><searchLink fieldCode="AU" term="%22Murphy+E%22">Murphy E</searchLink>; National Hospital for Neurology and Neurosurgery, London, UK.<br /><searchLink fieldCode="AU" term="%22Reisewitz+P%22">Reisewitz P</searchLink>; BioMarin Pharmaceutical Inc., Novato, CA, USA.<br /><searchLink fieldCode="AU" term="%22Mitchell+JJ%22">Mitchell JJ</searchLink>; McGill University Health Centre, Montreal, Quebec, Canada.
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  Data: <searchLink fieldCode="JN" term="%22101266602%22">Orphanet journal of rare diseases</searchLink> [Orphanet J Rare Dis] 2025 Oct 30; Vol. 20 (1), pp. 548. <i>Date of Electronic Publication: </i>2025 Oct 30.
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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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