In 2018, the research group of prof. Marjo van der Knaap demonstrated a beneficial effect of Guanabenz, an old and well-known FDA-approved drug for hypertension, in the mouse model for VWM (see publication).
After more than four years of fundraising, clinical trial design drafting, ethical review, drug production, and COVID-19 related delay, the Guanabenz clinical trial for VWM opened for patient enrollment on June 1, 2021. This is the very first clinical trial for VWM, and a big step towards therapy development. We are pleased to announce that the first three VWM patients have been enrolled for the study up to date, and more patients will follow soon.
We are looking forward to the study results in due time, and are excited about making progress to achieve our VWM consortium mission: enabling and accelerating therapeutics for Vanishing White Matter that is accessible and affordable for all patients.
For more information about the Guanabenz clinical trial, visit our clinical trial page.