U.S., Dec. 24 -- ClinicalTrials.gov registry received information related to the study (NCT07300397) titled 'Single Patient Investigational Treatment for Cree Leukoencephalopathy' on Nov. 26.
Brief Summary: Cree Leukoencephalopathy (CLE) is a very rare and severe brain disease that mainly affects members of the Cree communities in Northern Quebec. It causes the white matter of the brain-the part that helps nerves communicate-to slowly break down. As the disease progresses, children develop serious neurological problems that worsen over time and, sadly, lead to early death. At the moment, there are no effective treatments for CLE. The disease is caused by a single genetic change in the EIF2B5 gene, the same gene involved in another related ...