Toileting Abilities Survey as a surrogate outcome measure for cognitive function : findings from neuronopathic mucopolysaccharidosis II patients treated with idursulfase and intrathecal idursulfase

dc.contributor.authorHogan, Melissa J.
dc.contributor.authorStephens, Kim
dc.contributor.authorSmith, Erin
dc.contributor.authorJalazo, Elizabeth R.
dc.contributor.authorHendriksz, Christian J.
dc.contributor.authorEdwards, Lloyd J.
dc.contributor.authorBjoraker, Kendra J.
dc.date.accessioned2020-12-23T07:24:37Z
dc.date.available2020-12-23T07:24:37Z
dc.date.issued2020-12
dc.description.abstractAn 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.en_ZA
dc.description.departmentPaediatrics and Child Healthen_ZA
dc.description.librarianpm2020en_ZA
dc.description.urihttp://www.elsevier.com/locate/ymgmren_ZA
dc.identifier.citationHogan, M.J., Stephens, K., Smith, E. et al. 2020, '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, vol. 25, art. 100669, pp. 1-3.en_ZA
dc.identifier.issn2214-4269 (online)
dc.identifier.other10.1016/j.ymgmr.2020.100669
dc.identifier.urihttp://hdl.handle.net/2263/77480
dc.language.isoenen_ZA
dc.publisherElsevieren_ZA
dc.rights© 2020 Published by Elsevier Inc. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).en_ZA
dc.subjectHunter syndromeen_ZA
dc.subjectMucopolysaccharidosisen_ZA
dc.subjectIntrathecalen_ZA
dc.subjectIdursulfaseen_ZA
dc.subjectActivities of daily livingen_ZA
dc.subjectToiletingen_ZA
dc.subjectToileting abilities survey (TAS)en_ZA
dc.titleToileting Abilities Survey as a surrogate outcome measure for cognitive function : findings from neuronopathic mucopolysaccharidosis II patients treated with idursulfase and intrathecal idursulfaseen_ZA
dc.typeArticleen_ZA

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