A Scale to Assess Activities of Daily Living in Pantothenate Kinase-Associated Neurodegeneration.

ABSTRACT:

Objective

Pantothenate kinase-associated neurodegeneration (PKAN) is an autosomal-recessive, neurodegenerative disorder with a mixed-motor phenotype caused by a defective PanK2 enzyme, for which there are few adequate treatment options. Clinimetrically sound measures of patient-reported outcomes are necessary to facilitate therapeutic development for this debilitating disease. This study's objective was to develop such a scale and assess its clinimetric properties.

Methods

A conceptually driven, iterative, content development process incorporating input from experts, caregivers, and patients was used. Scale items were initially adapted from the Unified Parkinson's Disease Rating Scale (UPDRS) Part II resulting in the 12-item Pantothenate Kinase-Associated Neurodegeneration A

SUBMITTER: Marshall RD 

PROVIDER: S-EPMC6384181 | biostudies-literature | 2019 Feb

REPOSITORIES: biostudies-literature