Neurofilament light chain (NfL) as a biomarker of hereditary transthyretin-mediated amyloidosis

Paul Nioi, Simina Ticau, Gautham V. Sridharan, Shira Tsour, William L. Cantley, Amy Chan, Jason A. Gilbert, David Erbe, Emre Aldinc, Mary M. Reilly, David Adams, Michael Polydefkis, Kevin Fitzgerald & Akshay Vaishnaw
To identify changes in the proteome associated with onset and progression of ATTRv amyloidosis, we performed an observational, case-controlled study which compared proteomes of patients with ATTRv amyloidosis and healthy controls. Plasma levels of >1,000 proteins were measured in patients with ATTRv amyloidosis with polyneuropathy who received either placebo or patisiran in the APOLLO study and in healthy controls. The impact of patisiran on the time profile of each protein was determined by linear mixed...
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