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. |
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| 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 |
| ISSN: | 2949-7744 |
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| DOI: | 10.1016/j.gimo.2025.103428 |
