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). |
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| 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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| ISSN: | 1750-1172 |
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| DOI: | 10.1186/s13023-025-04064-w |
