With increased insight into Vanishing white matter and potential drugs in early development, it is time to start thinking about clinical trial design. Considering several challenges (such as low numbers of patients, unpredictable disease course, absence of intermediate biomarkers), smart clinical trial design and close collaboration with all stakeholders is key. In the first VWM consortium paper together with patient associations, we have formulated recommendations for enhanced diagnosis, drug trial setup, and patient selection, based on our expert evaluation of molecular, laboratory, and clinical data.
This is an important step in the drug development landscape for VWM. Please read our published paper in Neurology Genetics here.