Clinical Trial Simulations Based on Genetic Stratification and the Natural History of a Functional Outcome Measure in Creutzfeldt-Jakob Disease

IMPORTANCE: A major challenge for drug development in neurodegenerative diseases is that adequately powered efficacy studies with meaningful end points typically require several hundred participants and long durations. Prion diseases represent the archetype of brain diseases caused by protein misfol...

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Pubblicato in:JAMA Neurol
Autori principali: Mead, Simon, Burnell, Matthew, Lowe, Jessica, Thompson, Andrew, Lukic, Ana, Porter, Marie-Claire, Carswell, Christopher, Kaski, Diego, Kenny, Janna, Mok, Tze How, Bjurstrom, Nina, Franko, Edit, Gorham, Michele, Druyeh, Ronald, Wadsworth, Jonathan D. F., Jaunmuktane, Zane, Brandner, Sebastian, Hyare, Harpreet, Rudge, Peter, Walker, A. Sarah, Collinge, John
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2016
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5701731/
https://ncbi.nlm.nih.gov/pubmed/26902324
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1001/jamaneurol.2015.4885
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