Pediatric pharmacokinetics and pharmacodynamics of guanabenz for the treatment of vanishing white matter (opens in new tab)

Vanishing White Matter (VWM) is a leukodystrophy with cystic cerebral white matter degeneration, resulting in neurological decline and premature death. There is currently no treatment. Guanabenz, a central α2-adrenergic receptor agonist, ameliorates the disease in VWM mouse models. We aimed to evaluate the validity of a translational guanabenz pediatric pharmacokinetic model, to explore the concentration-toxicity association, and to estimate brain guanabenz concentrations using a biomarker of...