Association of CAG Repeats With Long-term Progression in Huntington Disease.

ABSTRACT:

Importance

In Huntington disease (HD), mutation severity is defined by the length of the CAG trinucleotide sequence, a well-known predictor of clinical onset age. The association with disease trajectory is less well characterized. Quantifiable summary measures of trajectory applicable over decades of early disease progression are lacking. An accurate model of the age-CAG association with early progression is critical to clinical trial design, informing both sample size and intervention timing.

Objective

To succinctly capture the decades-long early progression of HD and its dependence on CAG repeat length.

Design, setting, and participants

Prospective study at 4 academic HD treatment and research centers. Participants were the combined sample from the TRACK-HD and Trac

SUBMITTER: Langbehn DR 

PROVIDER: S-EPMC6692683 | biostudies-literature | 2019 Nov

REPOSITORIES: biostudies-literature