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

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...

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Autores principales: Hogan, Melissa J., Stephens, Kim, Smith, Erin, Jalazo, Elizabeth R., Hendriksz, Christian J., Edwards, Lloyd J., Bjoraker, Kendra J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2020
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7578548/
https://www.ncbi.nlm.nih.gov/pubmed/33101985
http://dx.doi.org/10.1016/j.ymgmr.2020.100669
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author Hogan, Melissa J.
Stephens, Kim
Smith, Erin
Jalazo, Elizabeth R.
Hendriksz, Christian J.
Edwards, Lloyd J.
Bjoraker, Kendra J.
author_facet Hogan, Melissa J.
Stephens, Kim
Smith, Erin
Jalazo, Elizabeth R.
Hendriksz, Christian J.
Edwards, Lloyd J.
Bjoraker, Kendra J.
author_sort Hogan, Melissa J.
collection PubMed
description 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.
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spelling pubmed-75785482020-10-23 Toileting Abilities Survey as a surrogate outcome measure for cognitive function: Findings from neuronopathic mucopolysaccharidosis II patients treated with idursulfase and intrathecal idursulfase Hogan, Melissa J. Stephens, Kim Smith, Erin Jalazo, Elizabeth R. Hendriksz, Christian J. Edwards, Lloyd J. Bjoraker, Kendra J. Mol Genet Metab Rep Research Paper 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. Elsevier 2020-10-21 /pmc/articles/PMC7578548/ /pubmed/33101985 http://dx.doi.org/10.1016/j.ymgmr.2020.100669 Text en © 2020 Published by Elsevier Inc. http://creativecommons.org/licenses/by-nc-nd/4.0/ This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle Research Paper
Hogan, Melissa J.
Stephens, Kim
Smith, Erin
Jalazo, Elizabeth R.
Hendriksz, Christian J.
Edwards, Lloyd J.
Bjoraker, Kendra J.
Toileting Abilities Survey as a surrogate outcome measure for cognitive function: Findings from neuronopathic mucopolysaccharidosis II patients treated with idursulfase and intrathecal idursulfase
title Toileting Abilities Survey as a surrogate outcome measure for cognitive function: Findings from neuronopathic mucopolysaccharidosis II patients treated with idursulfase and intrathecal idursulfase
title_full Toileting Abilities Survey as a surrogate outcome measure for cognitive function: Findings from neuronopathic mucopolysaccharidosis II patients treated with idursulfase and intrathecal idursulfase
title_fullStr Toileting Abilities Survey as a surrogate outcome measure for cognitive function: Findings from neuronopathic mucopolysaccharidosis II patients treated with idursulfase and intrathecal idursulfase
title_full_unstemmed Toileting Abilities Survey as a surrogate outcome measure for cognitive function: Findings from neuronopathic mucopolysaccharidosis II patients treated with idursulfase and intrathecal idursulfase
title_short Toileting Abilities Survey as a surrogate outcome measure for cognitive function: Findings from neuronopathic mucopolysaccharidosis II patients treated with idursulfase and intrathecal idursulfase
title_sort toileting abilities survey as a surrogate outcome measure for cognitive function: findings from neuronopathic mucopolysaccharidosis ii patients treated with idursulfase and intrathecal idursulfase
topic Research Paper
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7578548/
https://www.ncbi.nlm.nih.gov/pubmed/33101985
http://dx.doi.org/10.1016/j.ymgmr.2020.100669
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