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Toileting Abilities Survey as a surrogate outcome measure for cognitive function: Findings from neuronopathic mucopolysaccharidosis II patients treated with idursulfase and intrathecal idursulfase
Molecular Genetics and Metabolism Reports ( IF 1.8 ) Pub Date : 2020-10-21 , DOI: 10.1016/j.ymgmr.2020.100669
Melissa J. Hogan , Kim Stephens , Erin Smith , Elizabeth R. Jalazo , Christian J. Hendriksz , Lloyd J. Edwards , Kendra J. Bjoraker

An outcome measure of toileting skills, the Toileting Abilities Survey or TAS, with sensitivity to detect change in a neurodegenerative disorder such as MPS II, was developed. The TAS was used in a research study of patients (n = 86) with the neuronopathic form of MPS II to measure treatment benefit of intrathecal idursulfase. Treatment with idursulfase and intrathecal idursulfase is associated with significantly higher individual and overall toileting skills versus treatment with idursulfase alone.



中文翻译:

厕所能力调查作为认知功能的替代指标:来自异硫脲酶和鞘内异硫脲酶治疗的神经性黏膜多糖贮积症II患者的发现

开发了一种衡量坐便技巧的结果指标,即“坐便能力调查”或TAS,它能够检测出神经退行性疾病(例如MPS II)的变化。TAS用于 对神经病变形式MPS II的患者(n = 86)进行的一项研究研究,以测量鞘内注射异硫氰酸酯的治疗效果。与仅使用艾杜硫酶治疗相比,使用艾杜硫酶和鞘内注射艾杜硫酶治疗可显着提高个人和整体洗手技能。

更新日期:2020-10-29
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