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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Published in:JAMA Neurol
Main Authors: 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
Format: Artigo
Language:Inglês
Published: 2016
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Online Access: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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