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

Abstract excerpt

Objective To determine whether preventive trials in genetic prion disease could be designed to follow presymptomatic mutation carriers to onset of disease. Methods We assembled age at onset or death data from 1,094 individuals with high penetrance mutations in the prion protein gene ( PRNP ) in order to generate survival and hazard curves and test for genetic modifiers of age at onset. We used formulae and...

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