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Findings from the Morquio A Registry Study (MARS) after 6 Years: Long-term Outcomes of MPS IVA Patients Treated with Elosulfase Alfa

John J. Mitchell,Barbara K. Burton,Michael B. Bober,Philippe M. Campeau,Shelda Cohen,Sara Dosenovic,Carolyn Ellaway,Kaustuv Bhattacharya,Nathalie Guffon,David Hinds,Alice Lail,Shuan-Pei Lin,Martin Magner,Julian Raiman, Liat Schwartz-Sagi,Karolina M. Stepien

Molecular Genetics and Metabolism（2022）

引用 2|浏览18
关键词
Elosulfase alfa,enzyme replacement therapy,mucopolysaccharidosis IVA,Morquio A syndrome&nbsp,registry
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