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No recognized specific surrogate marker currently can facilitate diagnosis or assessment of prognosis in amyotrophic lateral sclerosis (ALS). This problem has long been a major impediment in the conduct of clinical trials and, indeed, in clinical practice.
Recent studies of protein expression in CSF and blood have addressed this problem. The results inevitably consist of the identification of large numbers of proteins differing in concentration in patients with ALS vs normal control subjects or, more appropriately, patients with other neurologic disorders. This has led to the realization that there are many technical and clinical limitations in these studies.1 Early studies of proteins in CSF using proteomics technology set out to characterize the protein constituents of CSF in neurodegenerative disorders, such as ALS and Alzheimer dementia, and also in inflammatory disease, especially multiple sclerosis. CSF is a fluid with slow circulation characteristics, and it has long been know that its characteristics differ by location, that is, in samples taken at lumbar puncture, cisternal puncture, or in ventricular specimens. The possibility of differing concentrations of …
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