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Long-term outcomes of elosulfase alfa enzyme replacement therapy in adults with MPS IVA: a sub-analysis of the Morquio A Registry Study (MARS).

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Title: Long-term outcomes of elosulfase alfa enzyme replacement therapy in adults with MPS IVA: a sub-analysis of the Morquio A Registry Study (MARS).
Authors: Stepien, Karolina M.1 (AUTHOR) karolina.stepien@nca.nhs.uk, Burton, Barbara K.2 (AUTHOR), Bober, Michael B.3 (AUTHOR), Campeau, Philippe M.4 (AUTHOR), Ellaway, Carolyn5 (AUTHOR), Bhattacharya, Kaustuv5 (AUTHOR), Guffon, Nathalie6 (AUTHOR), Hinds, David7 (AUTHOR), Hunt, Abigail7 (AUTHOR), Lail, Alice7 (AUTHOR), Lin, Shuan-Pei8 (AUTHOR), Magner, Martin9 (AUTHOR), Murphy, Elaine10 (AUTHOR), Reisewitz, Pascal7 (AUTHOR), Mitchell, John J.11 (AUTHOR)
Source: Orphanet Journal of Rare Diseases. 10/30/2025, Vol. 20 Issue 1, p1-10. 10p.
Database: Academic Search Ultimate
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Description
ISSN:17501172
DOI:10.1186/s13023-025-04064-w