Disease Progression in Huntington Disease: An Analysis of Multiple Longitudinal Outcomes.

ABSTRACT:

Background

Critical to discovering targeted therapies for Huntington disease (HD) are validated methods that more precisely predict when clinical outcomes occur for different patient profiles.

Objective

To more precisely predict the probability of when motor diagnosis (diagnostic confidence level 4) on the Unified Huntington's Disease Rating Scale (UHDRS), cognitive impairment (two or more neuropsychological scores on the UHDRS were 1.5 standard deviations below normative means) and Stage II Total Functional Capacity (TFC) first occur by accounting for dependencies between these outcomes.

Methods

Adult premanifest participants with ≥36 CAG repeats were selected from multi-center, longitudinal, observational studies: Prospective Huntington At Risk Observational Study (

SUBMITTER: Garcia TP 

PROVIDER: S-EPMC9012322 | biostudies-literature | 2018

REPOSITORIES: biostudies-literature