A Study to Explore the Safety, Tolerability, Pharmacokinetic Profile, and Potential Efficacy of Guanabenz in Patients With Early Childhood Onset Vanishing White Matter (VWM)

Pure Amsterdam UMC 2026 Dataset Open access

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Authors: Marjo S. van der Knaap
DOI: 10.34894/4i9upp
Cited by: 0
References: 1

Abstract

This clinical trial dataset comprises data from 33 patients with Vanishing White Matter who are treated with Guanabenz.

Fields of Study & Subjects

Metabolism and Genetic Disorders Pharmaceutical studies and practices Fetal and Pediatric Neurological Disorders

Publication details

Journal / source
Pure Amsterdam UMC
Publication date
4 February 2026
Document type
Dataset