Article

Age of onset in genetic prion disease and the design of preventive clinical trials

2018-08-29

Abstract excerpt

Regulatory agencies worldwide have adopted programs to facilitate drug development for diseases where the traditional approach of a randomized trial with a clinical endpoint is expected to be prohibitively lengthy or difficult. Here we provide quantitative evidence that this criterion is met for the prevention of genetic prion disease. We assemble age of onset or death data from N =1,094 individuals with high pen...

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Literature Corpus work: 0e11e4cf-02e2-58ce-b22c-4efc2af1db7c
DOI: 10.1101/401406

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