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[HEALTH] · Netherlands · 6 sources

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Amsterdam UMC study finds blood pressure drug may slow rare childhood brain disease

A study led by Amsterdam University Medical Centers (Amsterdam UMC) suggests that guanabenz, an existing blood pressure medication, may slow the progression of vanishing white matter (VWM) disease. VWM is a rare, hereditary neurodegenerative disorder that primarily affects children between the ages of one and six, causing a progressive loss of motor and cognitive functions that can lead to death.

Published in ‘The Lancet Neurology’, the research monitored children with VWM receiving guanabenz over a three-year period and compared them to a group of 66 children with similar disease severity from an international registry who did not receive the drug. The study found that children treated with guanabenz became dependent on wheelchairs less frequently and at a slower rate than the comparison group. Notably, no children receiving guanabenz died during the study period, whereas five deaths occurred in the untreated group.

While the medication is not a cure, researchers noted that side effects—including hallucinations, drowsiness, constipation, and low blood pressure—were primarily observed during the first few months of treatment. Most children tolerated the medication well after four to six months.

Entities

Amsterdam UMC · Amsterdam University Medical Centers · Guanabenz · Marjo van der Kanap · Marjo van der Knaap · The Lancet Neurology · Vanishing White Matter disease

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