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Plasma Proteome Analysis of Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis Establishes Neurofilament Light Chain (NfL) as a Biomarker of Disease and Treatment Response
2019-11-08
neurology
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BackgroundHereditary transthyretin-mediated (hATTR) amyloidosis is a rare, progressively debilitating, and fatal disease caused by deposition of aggregated transthyretin amyloid in multiple organs and tissues. Highly variable disease penetrance has made it difficult to predict disease onset and progression. Clinically validated, non-invasive plasma biomarkers may facilitate earlier diagnosis and aid monitoring of disease progression. MethodsPlasma levels of >1000 proteins were measured in patie...
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