As we enter an era of secondary prevention trials to treat or prevent neurodegenerative diseases, the need to optimize clinical trial design and maximize efficiency is paramount. Biomarkers will be central to such efforts, wherein treatment is to be administered only to individuals affected by the targeted abnormalities and when symptoms are mild or absent. Cerebrospinal fluid (CSF) biomarkers will play a particularly vital role because they can provide a direct, real-time, changeable reflection of the physiology and biochemistry of the brain and spinal cord.
Perrin RJ. Cerebrospinal Fluid Biomarkers for Clinical Trials: Why Markers for Differential Diagnosis Are Important. Arch Neurol. 2012;69(11):1407–1408. doi:10.1001/archneurol.2012.2353
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