Survival End Points for Huntington Disease Trials Prior to a Motor Diagnosis.

Abstract

Importance Predictive genetic testing in Huntington disease (HD) enables therapeutic trials in HTT gene expansion mutation carriers prior to a motor diagnosis. Progression-free survival (PFS) is the composite of a motor diagnosis or a progression event, whichever comes first. Objective To determine if PFS provides feasible sample sizes for trials with… (More)
DOI: 10.1001/jamaneurol.2017.2107

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