Existing Blood Pressure Drug Shows Promise in Slowing Rare Childhood Brain Disease Progression

A study led by Amsterdam University Medical Centers, published in The Lancet Neurology, suggests that guanabenz, an existing blood pressure medication, may slow the progression of vanishing white matter (VWM), a rare and often fatal hereditary neurodegenerative disorder affecting children. Researchers observed that children with VWM treated with guanabenz became dependent on wheelchairs less frequently and less rapidly compared to an untreated comparison group. No deaths occurred in the treated group during the three-year study period, while five deaths were reported in the comparison group. Side effects were mainly observed in the initial months of treatment and were generally well-tolerated thereafter.