U.S., Dec. 10 -- ClinicalTrials.gov registry received information related to the study (NCT07272525) titled 'Research Study for Single-Patient Treatment of Cree Leukoencephalopathy/Vanishing White Matter Disease' on Oct. 11, 2024.
Brief Summary: Cree Leukoencephalopathy (CLE) is a rare and fatal neurodegenerative disorder predominantly affecting the Cree population in Northern Quebec. Characterized by progressive white matter degeneration, this condition leads to severe neurological impairment and decline, leading to premature death. Despite its significant impact on the affected population, there are currently no effective treatments for CLE. CLE is caused by a single founder pathogenic variant in the EIF2B5 gene and is therefore allelic ...