Age at onset in genetic prion disease and the design of preventive clinical trials.
Eric Vallabh MinikelSonia M VallabhMargaret C OrsethJean-Philippe BrandelStéphane HaïkJean-Louis LaplancheInga ZerrPiero ParchiSabina CapellariJiri SafarJanna KennyJamie C FongLeonel T TakadaClaudia PontoPeter HermannTobias KnipperChristiane StehmannTetsuyuki KitamotoRyusuke AeTsuyoshi HamaguchiNobuo SanjoTadashi TsukamotoHidehiro MizusawaSteven J CollinsRoberto ChiesaIgnazio RoiterJesús de Pedro-CuestaMiguel CaleroMichael D GeschwindMasahito YamadaYosikazu NakamuraSimon MeadPublished in: Neurology (2019)
The characterization of biomarkers suitable to serve as surrogate endpoints will be essential for the prevention of genetic prion disease. Parameters such as longer trial duration, increased enrollment, and the use of historical controls in a postmarketing study could provide opportunities for subsequent determination of clinical benefit.