Experimental drug offered for devastating rare brain diseases
NCT ID NCT06594016
Summary
This program provided early access to an investigational drug called fosigotifator for people with vanishing white matter disease and Cree leukoencephalopathy—two rare, serious brain disorders. It was designed to make the treatment available to eligible patients before it received full regulatory approval. Doctors could enroll patients if they determined the potential benefits outweighed the risks based on individual medical history.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for VANISHING WHITE MATTER are added.
By submitting, you agree to our Terms of use
Conditions
Explore the condition pages connected to this study.