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

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 determin...

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Vydáno v:JAMA Neurol
Hlavní autoři: Long, Jeffrey D., Mills, James A., Leavitt, Blair R., Durr, Alexandra, Roos, Raymund A., Stout, Julie C., Reilmann, Ralf, Landwehrmeyer, Bernhard, Gregory, Sarah, Scahill, Rachael I., Langbehn, Douglas R., Tabrizi, Sarah J.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Medical Association 2017
Témata:
Original Investigation
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5710578/
https://ncbi.nlm.nih.gov/pubmed/28975278
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1001/jamaneurol.2017.2107
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