Finding a cure for
Vanishing White Matter
About VWM
Guanabenz clinical trial
enrollment started
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Mission statement

Our mission is to enable and accelerate therapeutics for Vanishing White Matter, aiming at accessibility and affordability for all patients. To this purpose, we established an international collaboration, which engages partners to provide unbiased analyses of different therapeutics, using smart trial design to address recruitment challenges of this rare disease. We work together with a number of partner organizations, including academic institutes, biotech and pharma companies, and patient and family organizations.

Current Trial

Guanabenz trial for Vanishing White Matter

The Amsterdam Leukodystrophy Center at the Amsterdam University Medical Centers (Amsterdam UMC) is conducting a clinical trial in children with Vanishing White Matter (VWM). In this trial, we want to evaluate whether a drug called Guanabenz is effective in slowing progression, stabilizing, or even improving the brain white matter abnormalities in VWM.

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Consortium Members
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