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

Abstract Background Mucopolysaccharidosis (MPS) IVA is a rare disease with substantial, multisystemic morbidity. We assessed real-world safety and effectiveness of the enzyme replacement therapy (ERT) elosulfase alfa in patients with MPS IVA in the multinational, observational Morquio A Registry Stu...

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Principais autores:	Karolina M. Stepien, Barbara K. Burton, Michael B. Bober, Philippe M. Campeau, Carolyn Ellaway, Kaustuv Bhattacharya, Nathalie Guffon, David Hinds, Abigail Hunt, Alice Lail, Shuan-Pei Lin, Martin Magner, Elaine Murphy, Pascal Reisewitz, John J. Mitchell
格式:	Artigo
語言:	Inglês
出版:	BMC 2025-10-01
叢編:	Orphanet Journal of Rare Diseases
主題:	Elosulfase alfa Mucopolysaccharidosis IVA Clinical outcomes Patient-reported outcomes Real-world evidence
在線閱讀:	https://doi.org/10.1186/s13023-025-04064-w
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