Vanishing White Matter research hub

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

This study is looking at a new medicine called Guanabenz for children with a rare brain condition called Vanishing White Matter (VWM). Researchers want to see if it's safe, how the body handles it, and if it could help treat the condition.