Open AccessSurvival End Points for Huntington Disease Trials Prior to a Motor Diagnosis
Author(s) -
Jeffrey D. Long,
James A. Mills,
Blair R. Leavitt,
Alexandra Dürr,
Raymund A.C. Roos,
Julie C. Stout,
Ralf Reilmann,
G. Bernhard Landwehrmeyer,
Sarah Gregory,
Rachael I. Scahill,
Douglas R. Langbehn,
Sarah J. Tabrizi
Publication year - 2017
Publication title -
jama neurology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 5.298
H-Index - 231
eISSN - 2168-6157
pISSN - 2168-6149
DOI - 10.1001/jamaneurol.2017.2107
Subject(s) - cohort , medicine , cohort study , clinical trial
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.
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