Hogan, Melissa J.Stephens, KimSmith, ErinJalazo, Elizabeth R.Hendriksz, Christian J.Edwards, Lloyd J.Bjoraker, Kendra J.2020-12-232020-12-232020-12Hogan, 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. 1-3.2214-4269 (online)10.1016/j.ymgmr.2020.100669http://hdl.handle.net/2263/77480An 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© 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/).Hunter syndromeMucopolysaccharidosisIntrathecalIdursulfaseActivities of daily livingToiletingToileting abilities survey (TAS)Article