Clinical expert opinion on the role of elosulfase alfa in non-ambulatory individuals with Morquio A syndrome

This explorative study offers valuable insights into drivers of ERT use in non-ambulatory patients with Morquio A, its potential benefits, and strategies for monitoring treatment outcomes in clinical practice. These findings may guide patient management, optimize treatment, and inform future research for this population.

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Publication Details

Journal
Molecular Genetics and Metabolism Reports
Published
2026-01-21
DOI
https://doi.org/10.1016/j.ymgmr.2026.101290
Primary Topic
Lysosomal Storage Disorders Research
Type
article
Field-Weighted Citation Impact
0.00
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article

Clinical expert opinion on the role of elosulfase alfa in non-ambulatory individuals with Morquio A syndrome

Karolina M. Stępień, P. Campeau, Nicole Muschol, Carolina FM De Souza et al.
Molecular Genetics and Metabolism Reports
Lysosomal Storage Disorders Research
article

Clinical expert opinion on the role of elosulfase alfa in non-ambulatory individuals with Morquio A syndrome

Karolina M. Stępień, P. Campeau, Nicole Muschol, Carolina FM De Souza, Serap Hatice Kalkanoğlu Sivri, Martha Solano, Barbara K. Burton, Christina Lampe, Roberto Giugliani
article en

Abstract

This explorative study offers valuable insights into drivers of ERT use in non-ambulatory patients with Morquio A, its potential benefits, and strategies for monitoring treatment outcomes in clinical practice. These findings may guide patient management, optimize treatment, and inform future research for this population.

Molecular Genetics and Metabolism ReportsVol. 46
Northwestern University (US), Lurie Children's Hospital (US), Universidade Federal do Rio Grande do Sul (BR), Universität Hamburg (DE), Centre Hospitalier Universitaire Sainte-Justine (CA), Hospital de Clínicas de Porto Alegre (BR), Hospices Civils de Lyon (FR), Lysosomal and Rare Disorders Research and Treatment Center (US), University Medical Center Hamburg-Eppendorf (DE), Salford Royal Hospital (GB), Hacettepe University Hospital (TR), Institute of Cardiology (CO)
Good health and well-being
Openalex Percentile: Top 83%
Lysosomal Storage Disorders Research
DOIOpen Access PDFOpenAlex
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Clinical expert opinion on the role of elosulfase alfa in non-ambulatory individuals with Morquio A syndrome — Karolina M. Stępień, P. Campeau, et al. · Molecular Genetics and Metabolism Reports (2026) | TGRS Research Map | TGRS