Trusted Resources: Education

Scientific literature and patient education texts

Analysis of Cognitive Ability and Adaptive Behavior Assessment Tools Used in an Observational Study of Patients With Mucopolysaccharidosis II

key information

source: Orphanet journal of rare diseases

year: 2021

authors: Yee KS,Wu Y,Harrington M,Waisbren SE


Mucopolysaccharidosis II (MPS II) is a rare lysosomal storage disease characterized by cognitive impairment in most patients. This post hoc analysis evaluated changes in cognitive function, adaptive behavior and functional outcomes in patients with neuronopathic MPS II over time. Fifty-five children with MPS II were enrolled in a 24-month observational study (NCT01822184). The Differential Ability Scales, second edition (DAS-II; early years battery for ages 2 years 6 months to 6 years 11 months, school age battery for ages 7 years to 17 years 11 months), Vineland Adaptive Behavior Scales, second edition (VABS-II) and the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) were performed at baseline and 3-month intervals over 2 years. A subgroup of 38 children with a DAS-II General Conceptual Ability (GCA) score of 55-85 (below average-very low abilities) at any time during the study were included in this analysis.

organization: Takeda Development Center Americas, Inc., Cambridge, MA, USA.

DOI: 10.1186/s13023-021-02118-3

read more