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）

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摘要

MARS is the longest and largest observational study of MPS IVA patients to date, with a heterogenous population that is representative of the MPS IVA population overall. Data collected over the first 6 years of MARS provide real-world evidence for long-term stabilization of endurance and respiratory function among ERT-treated patients, with no new safety concerns identified.

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关键词

Elosulfase alfa,enzyme replacement therapy,mucopolysaccharidosis IVA,Morquio A syndrome,registry

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