Improvements in quality of life outcomes among individuals with MPS IVA treated with elosulfase alfa: Results from the Morquio A Registry Study (MARS)

Philippe M. Campeau; Barbara K. Burton; Carolyn Ellaway; Nathalie Guffon; Derralynn A. Hughes; Abigail Hunt; Alice E. Lail; Hsiang-Yu Lin; Shuan Pei Lin; Martin Magner; John J. Mitchell; Pascal Reisewitz; Karolina M. Stępień

doi:10.1016/j.ymgme.2025.109352

Improvements in quality of life outcomes among individuals with MPS IVA treated with elosulfase alfa: Results from the Morquio A Registry Study (MARS)

Molecular Genetics and Metabolism 2026

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Authors: Philippe M. Campeau, Barbara K. Burton, Carolyn Ellaway, Nathalie Guffon, Derralynn A. Hughes, Abigail Hunt, Alice E. Lail, Hsiang-Yu Lin, Shuan Pei Lin, Martin Magner, John J. Mitchell, Pascal Reisewitz

+1 more

Karolina M. Stępień

DOI: 10.1016/j.ymgme.2025.109352

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Proteoglycans and glycosaminoglycans research

Publication details

Journal / source: Molecular Genetics and Metabolism
Volume: 147
Issue: 2
Pages: 109352–109352
Publication date: 1 February 2026
Language: EN