Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over time
Purpose: To assess the real-world effectiveness of enzyme replacement therapy (ERT; elosulfase alfa) on endurance in the treatment of mucopolysaccharidosis type IVA (MPS IVA) using cross-sectional data. Methods: The 6-minute walk test (6MWT) distances of ERT-treated and untreated participants from t...
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| Автори: | , , , , , , |
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| Формат: | Artigo |
| Мова: | Inglês |
| Опубліковано: |
Elsevier
2025-01-01
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| Серія: | Genetics in Medicine Open |
| Предмети: | |
| Онлайн доступ: | http://www.sciencedirect.com/science/article/pii/S2949774425014670 |
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