Ontology highlight
ABSTRACT:
SUBMITTER: Hogan MJ
PROVIDER: S-EPMC7578548 | biostudies-literature | 2020 Dec
REPOSITORIES: biostudies-literature
Molecular genetics and metabolism reports 20201021
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 (<i>n</i> = 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 idursul ...[more]