Understanding the Outcomes Measures used in Huntington Disease Pharmacological Trials: A Systematic Review.

ABSTRACT:

Background

The identification of the gene mutation causing Huntington disease has raised hopes for new treatments to ease symptoms and slow functional decline. As such, there has been a push towards designing efficient pharmacological trials (i.e., drug trials), especially with regard to selecting outcomes measures that are both brief and sensitive to changes across the course of the disease, from subtle prodromal changes, to more severe end-stage changes.

Objectives

Recently, to aid in efficient development of new HD research studies, the National Institute of Neurological Disorders and Stroke (NINDS) published recommendations for measurement selection in HD. While these recommendations are helpful, many of the recommended measures have little published data in HD. As such,

SUBMITTER: Carlozzi NE 

PROVIDER: S-EPMC4217648 | biostudies-literature | 2014

REPOSITORIES: biostudies-literature