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Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over time.

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Title: Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over time.
Authors: Burton BK; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL., Stepien KM; Northern Care Alliance NHS Foundation Trust, Salford, United Kingdom., Campeau PM; CHU Sainte-Justine Research Center, Montreal, QC, Canada., Sutton J; BioMarin (UK) Limited, London, United Kingdom., Hunt A; BioMarin Pharmaceutical Inc, Novato, CA., Reisewitz P; BioMarin Pharmaceutical Inc, Novato, CA., Hinds D; BioMarin Pharmaceutical Inc, Novato, CA.
Source: Genetics in medicine open [Genet Med Open] 2025 Mar 29; Vol. 3, pp. 103428. Date of Electronic Publication: 2025 Mar 29 (Print Publication: 2025).
Publication Type: Journal Article
Journal Info: Publisher: Elsevier Inc Country of Publication: United States NLM ID: 9918734281906676 Publication Model: eCollection Cited Medium: Internet ISSN: 2949-7744 (Electronic) Linking ISSN: 29497744 NLM ISO Abbreviation: Genet Med Open Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
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PubType: Academic Journal
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  Data: Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over time.
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  Data: <searchLink fieldCode="AU" term="%22Burton+BK%22">Burton BK</searchLink>; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL.<br /><searchLink fieldCode="AU" term="%22Stepien+KM%22">Stepien KM</searchLink>; Northern Care Alliance NHS Foundation Trust, Salford, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Campeau+PM%22">Campeau PM</searchLink>; CHU Sainte-Justine Research Center, Montreal, QC, Canada.<br /><searchLink fieldCode="AU" term="%22Sutton+J%22">Sutton J</searchLink>; BioMarin (UK) Limited, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Hunt+A%22">Hunt A</searchLink>; BioMarin Pharmaceutical Inc, Novato, CA.<br /><searchLink fieldCode="AU" term="%22Reisewitz+P%22">Reisewitz P</searchLink>; BioMarin Pharmaceutical Inc, Novato, CA.<br /><searchLink fieldCode="AU" term="%22Hinds+D%22">Hinds D</searchLink>; BioMarin Pharmaceutical Inc, Novato, CA.
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  Data: <searchLink fieldCode="JN" term="%229918734281906676%22">Genetics in medicine open</searchLink> [Genet Med Open] 2025 Mar 29; Vol. 3, pp. 103428. <i>Date of Electronic Publication: </i>2025 Mar 29 (<i>Print Publication: </i>2025).
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Elsevier+Inc%22">Elsevier Inc </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>9918734281906676 <i>Publication Model: </i>eCollection <i>Cited Medium: </i>Internet <i>ISSN: </i>2949-7744 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2229497744%22">29497744 </searchLink><i>NLM ISO Abbreviation: </i>Genet Med Open <i>Subsets: </i>PubMed not MEDLINE
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        Value: 10.1016/j.gimo.2025.103428
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        Text: English
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              Text: 2025 Mar 29
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