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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Bibliographic Details

Published in:	Genetics in Medicine Open
Main Authors:	Barbara K. Burton, Karolina M. Stepien, Philippe M. Campeau, Jaim Sutton, Abigail Hunt, Pascal Reisewitz, David Hinds
Format:	Article
Language:	English
Published:	Elsevier 2025-01-01
Subjects:	6-minute walk test Cross-sectional Mucopolysaccharidosis IVA Multivariate analysis Registry
Online Access:	http://www.sciencedirect.com/science/article/pii/S2949774425014670