Pediatric pharmacokinetics and pharmacodynamics of guanabenz for the treatment of vanishing white matter

Abstract 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...

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Autori principali:	Linda G. Franken, Bart van Lieshout, Joshua Eugenio, Renate Verbeek, Marije Voermans, Elske van den Berg, Pierre Bet, Ron A. A. Mathôt, Dennis van der Laan, Truus E. M. Abbink, Marjo S. van der Knaap, Imke H. Bartelink
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	Nature Portfolio 2026-06-01
Serie:	Scientific Reports
Soggetti:	Vanishing white matter Guanabenz Pharmacokinetics Pharmacodynamics Paediatric
Accesso online:	https://doi.org/10.1038/s41598-026-47959-9
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