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- Irene Cortese, Gina Norato, Patrick R Harrington, Therri Usher, Ilaria Mainardi, Guillaume Martin-Blondel, Paola Cinque, Eugene O Major, and Virginia Sheikh.
- Experimental Immunotherapeutics Unit, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA. Electronic address: corteseir@ninds.nih.gov.
- Lancet Neurol. 2024 May 1; 23 (5): 534544534-544.
AbstractProgressive multifocal leukoencephalopathy is a rare but devastating demyelinating disease caused by the JC virus (JCV), for which no therapeutics are approved. To make progress towards addressing this unmet medical need, innovations in clinical trial design are needed. Quantitative JCV DNA in CSF has the potential to serve as a valuable biomarker of progressive multifocal leukoencephalopathy disease and treatment response in clinical trials to expedite therapeutic development, as do neuroimaging and other fluid biomarkers such as neurofilament light chain. Specifically, JCV DNA in CSF could be used in clinical trials as an entry criterion, stratification factor, or predictor of clinical outcomes. Insights from the investigation of candidate biomarkers for progressive multifocal leukoencephalopathy might inform approaches to biomarker development for other rare diseases.Copyright © 2024 Elsevier Ltd. All rights reserved.
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