Age of onset in genetic prion disease and the design of preventive clinical trials
Eric Vallabh Minikel, Sonia M. Vallabh, Margaret C. Orseth, Jean‐Philippe Brandel, Stéphane Haı̈k, Jean Laplanche, Inga Zerr, Piero Parchi, Sabina Capellari, Jiri Safar, Janna Kenny, Jamie C Fong, Leonel Tadao Takada, Claudia Ponto, Péter Hermann, , Simon Mead
This paper quantifies the challenges of designing preventive clinical trials for genetic prion disease due to high variability in age of onset, suggesting the need for biomarker-based trials.
Neurology · Jun 6, 2019Peer-reviewedNeuroscience