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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Detalles Bibliográficos
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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