Clinical Outcomes and Selection Criteria for Prodromal Huntington's Disease Trials.

ABSTRACT:

Background

Huntington's disease (HD) develops in individuals with extended cytosine-adenine-guanine (CAG) repeats within the huntingtin (HTT) gene, causing neurodegeneration and progressive motor and cognitive symptoms. The inclusion of mutant HTT carriers in whom overt symptoms are not yet fully manifest in therapeutic trials would enable the development of treatments that delay or halt the accumulation of significant disability.

Objectives

The present analyses assess whether screening prediagnosis (preHD) individuals based on a normalized prognostic index (PIN) score would enable the selection of prodromal preHD subjects in whom longitudinal changes in established outcome measures might provide robust signals. It also compares the relative statistical effect size of longit

SUBMITTER: Langbehn DR 

PROVIDER: S-EPMC7818458 | biostudies-literature | 2020 Dec

REPOSITORIES: biostudies-literature