Vanishing White Matter Disease
Vanishing White Matter disease (VWM) is a rare genetic disorder that affects the brain's white matter, leading to progressive neurological symptoms such as loss of motor skills, muscle stiffness, and cognitive decline.
We are exploring the safety and tolerability of Fosigotifator in adults and children with Vanishing White Matter Disease. This study aims to understand how the body handles this new treatment.
Health conditions and diseases that the clinical trial is designed to study and treat.
Vanishing White Matter disease (VWM) is a rare genetic disorder that affects the brain's white matter, leading to progressive neurological symptoms such as loss of motor skills, muscle stiffness, and cognitive decline.
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.