Pharmacology & ToxicologybioRxivPreprint
Bayesian modeling and simulation to inform rare disease drug development early decision-making: application to Duchenne muscular dystrophy
J. Lennie, J. T. Mondick, M. R. Gastonguay
This paper develops a Bayesian framework for early drug development decisions in rare diseases, showing higher accuracy than frequentist methods in Duchenne muscular dystrophy trials.
DOI 10.1101/2021.02.05.429907