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Publication in The Lancet Marks New Milestone for GuanaRep and VWM Research

September 3, 2026
In the mediapress release

The results of the VWM1 study on guanabenz have been published in The Lancet Neurology in August and have since received national attention. NOS reports on the results showing the potential of guanabenz, an existing blood pressure medicine, to slow the progression of vanishing white matter (VWM) disease, a rare and serious neurological disorder that primarily affects children.

The VWM1 study investigated whether guanabenz could slow disease progression in patients with VWM. The study found evidence that treatment with guanabenz can reduce the loss of neurological function associated with the disease, supporting its potential as a treatment for VWM. The findings provide an important clinical basis for the further development of guanabenz and represent a significant step forward for a disease for which treatment options remain very limited.
The publication marks an important milestone in the longstanding research into VWM led by Prof. Marjo van der Knaap and her team at Amsterdam UMC.
Amsterdam Academic Ventures (AAV) is proud to contribute to the next stage of this work through GuanaRep, the company established to advance the development and registration of guanabenz as a potential treatment for VWM.
The progress of GuanaRep demonstrates how socially responsible drug development can translate promising academic research into potential treatments, with the needs of patients at the centre.

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