Last hope drug trial for kids with fatal brain disease

This study gives one child with Cree Leukoencephalopathy (CLE) access to an experimental drug called fosigotifator. CLE is a rare, inherited brain disease that destroys white matter and leads to early death. The drug aims to slow or stop the damage, possibly easing symptoms and improving quality of life. The study will monitor the child closely to see if the benefits outweigh the risks.