Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome
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- 作者：Christian J. Hendriksza ; chris.hendriksz@srft.nhs.uk" class="auth_mail" title="E-mail the corresponding author ; Rossella Parinib ; Moeenaldeen D. AlSayedc ; Julian Raimand ; Roberto Giuglianie ; Martha L. Solano Villarrealf ; John J. Mitchellg ; Barbara K. Burtonh ; Norberto Guelberti ; Fiona Stewartj ; Derralynn A. Hughesk ; Kenneth I. Bergerl ; Peter Slasorm ; Robert Matousekm ; Elaina Jureckim ; Adam J. Shaywitzm ; Paul R. Harmatzn
- 关键词：3MSCT ; 3-minute stair climb test ; 6MWT ; 6-minute walk test ; AE ; adverse event ; ERT ; enzyme replacement therapy ; GALNS ; enzyme N-acetylgalactosamine-6-sulfatase ; IAR ; infusion-associated reaction ; IgE ; immunoglobulin E ; ITT ; intent-to-treat ; KS ; keratan sulfate ; MPP ; modified per-protocol ; MorCAP ; Morquio A Clinical Assessment Program ; NAb ; neutralizing antibody ; PBO ; placebo ; QOW ; every other week ; QW ; once weekly ; SAE ; serious adverse event ; TAb ; total antibody ; uKS ; urine keratan sulfate
- 刊名：Molecular Genetics and Metabolism
- 出版年：2016
- 出版时间：September-October 2016
- 年：2016
- 卷：119
- 期：1-2
- 页码：131-143
- 全文大小：1128 K
文摘
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Elosulfase alfa ERT associated with long-term endurance improvements in Morquio A.
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Improvements over 2 years differed from the decline seen in untreated subjects.
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Benefits were sustained regardless of baseline endurance, walking aid use, and age.
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Long-term treatment also caused a sustained reduction in urine keratan sulfate.
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No new or unexpected safety signals were identified with long-term ERT.