
Episode #6
Marjo van der Knaap on vanishing white matter
For decades, a diagnosis of vanishing white matter disease came with no real treatment — so how is an old blood-pressure pill offering hope? In this episode of In Conversation With…The Lancet Neurology, Deputy Editor Laura Hart speaks with Professor Marjo van der Knaap (Amsterdam UMC) about her clinical trial of guanabenz in children with the leukodystrophy vanishing white matter. They unpack the integrated stress response and eIF2B pathophysiology, why guanabenz moved from mouse models into a paediatric trial, the striking effect of age of onset, unexpected hallucinations as a side-effect, and where combination therapy with eIF2B activators could take the field. Essential listening for neurologists on leukodystrophy, rare paediatric neurological disease, and translating stress-response biology into treatment. Click here to read the full article: https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(26)00241-3/fulltext

