Methods of neurodevelopmental assessment in children with neurodegenerative disease: Sanfilippo syndrome.

ABSTRACT:

Objectives

(1) Develop a methodology for obtaining reliable cognitive and developmental data in children with neurodegenerative disease and cognitive impairment and in turn monitor disease state and treatment outcomes. (2) Demonstrate validity of age-equivalent scores.

Methods

We present guidelines for obtaining accurate test scores in low-functioning and behaviorally disruptive pediatric patients, followed by a method validation study: (1) using disease-specific protocols to assess salient aspects of the known phenotype, (2) selecting appropriate tests, (3) managing behavior, and (4) using age-equivalent scores on standardized tools. We used the Bayley Scales of Infant Development-III or Kaufman Assessment Battery for Children-II with a group of 25 children with mucopolysac

SUBMITTER: Delaney KA 

PROVIDER: S-EPMC4110329 | biostudies-literature | 2014

REPOSITORIES: biostudies-literature