ArticleGenetics in medicine open2025
Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over time.
Article in Genetics in medicine open, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
4 citing papers in PubMed.
- Early Versus Late Enzyme Replacement Therapy in Siblings With Morquio A Syndrome: Insights Into Therapeutic Timing.JIMD reports · 2026Article
- Long-term follow-up and response to elosulfase alfa in mucopolysaccharidosis type IVA: a single-center cohort from the Czech Republic.Orphanet journal of rare diseases · 2026Article
- Long-term Natural History and Elosulfase Alfa Treatment for Mucopolysaccharidosis Type IVA: A Single-Center Study in the Czech Republic.Research square · 2026Article
- Long-term outcomes of elosulfase alfa enzyme replacement therapy in adults with MPS IVA: a sub-analysis of the Morquio A Registry Study (MARS).Orphanet journal of rare diseases · 2025Article
Corrections and comments
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Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
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 the Morquio A Registry Study and Morquio A Clinical Assessment Program were described for age groups of interest (5 to <7 years, 9 to <11 years, 14 to <16 years, and 20 to <30 years). Linear and quantile univariate regression were performed to explore variables associated with 6MWT (ERT, sex, age, standing height, body weight, region, and race). Multivariate regression analyses were performed using covariates identified in univariate analyses ( Results: A total of 471 participants were included; baseline characteristics were similar within age groups. Median 6MWT distances were numerically greater in ERT-treated versus untreated participants within each age group. Quantile regression adjusting for multiple factors indicated a consistent trend of improved 6MWT with ERT treatment. Standing height was also associated with longer 6MWT in the multivariate quantile analysis, except in participants aged 5 to <7 years. Conclusion: This analysis assessed associations between ERT exposure and endurance to confirm the real-world, long-term effectiveness of elosulfase alfa in participants with MPS IVA.
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