Andrade C. A peculiar form of peripheral neuropathy: familiar atypical generalized amyloidosis with special involvement of the peripheral nerves. Brain. 1952;75(3):408–27.
Article CAS PubMed Google Scholar
Coelho T, Maurer MS, Suhr OB. THAOS – the transthyretin amyloidosis outcomes survey: initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis. Curr Med Res Opin. 2013;29(1):63–76.
Article CAS PubMed Google Scholar
Gonzalez-Lopez E, et al. Prognosis of transthyretin cardiac amyloidosis without heart failure symptoms. JACC CardioOncol. 2022;4(4):442–54.
Article PubMed PubMed Central Google Scholar
Grogan M, et al. Natural history of wild-type transthyretin cardiac amyloidosis and risk stratification using a novel staging system. J Am Coll Cardiol. 2016;68(10):1014–20.
Koike H, et al. Natural history of transthyretin Val30Met familial amyloid polyneuropathy: analysis of late-onset cases from non-endemic areas. J Neurol Neurosurg Psychiatry. 2012;83(2):152–8.
Mariani L-L, et al. Genotype–phenotype correlation and course of transthyretin familial amyloid polyneuropathies in France. Ann Neurol. 2015;78(6):901–16.
Article CAS PubMed PubMed Central Google Scholar
Sattianayagam PT, et al. Cardiac phenotype and clinical outcome of familial amyloid polyneuropathy associated with transthyretin alanine 60 variant. Eur Heart J. 2011;33(9):1120–7.
Robbins J. Transthyretin from discovery to now. Clin Chem Lab Med. 2002;40(12):1183–90.
Article CAS PubMed Google Scholar
Raghu P, Sivakumar B. Interactions amongst plasma retinol-binding protein, transthyretin and their ligands: implications in vitamin A homeostasis and transthyretin amyloidosis. Biochim Biophys Acta. 2004;1703(1):1–9.
Article CAS PubMed Google Scholar
Connelly S, et al. Structure-based design of kinetic stabilizers that ameliorate the transthyretin amyloidoses. Curr Opin Struct Biol. 2010;20(1):54–62.
Article CAS PubMed PubMed Central Google Scholar
Johnson SM, et al. Native state kinetic stabilization as a strategy to ameliorate protein misfolding diseases: a focus on the transthyretin amyloidoses. Acc Chem Res. 2005;38(12):911–21.
Article CAS PubMed Google Scholar
Coelho T, et al. Eplontersen for hereditary Transthyretin amyloidosis with polyneuropathy. JAMA. 2023;330(15):1448–58.
Article CAS PubMed PubMed Central Google Scholar
Hanson JLS, et al. Use of serum transthyretin as a prognostic indicator and predictor of outcome in cardiac amyloid disease associated with wild-type transthyretin. Circ Heart Fail. 2018;11(2):e004000.
Article CAS PubMed PubMed Central Google Scholar
Chan GG, Koch CM, Connors LH. Blood proteomic profiling in inherited (ATTRm) and acquired (ATTRwt) forms of transthyretin-associated cardiac amyloidosis. J Proteome Res. 2017;16(4):1659–68.
Article CAS PubMed Google Scholar
Cowan KJ, et al. Recommendations for Selection and Characterization of Protein Biomarker Assay Calibrator Material. AAPS j. 2017;19(6):1550–63.
Article CAS PubMed Google Scholar
Leary BA, et al. Bioanalytical platform comparison using a generic human IgG PK assay format. J Immunol Methods. 2013;397(1):28–36.
Article CAS PubMed Google Scholar
Lee JW, et al. Fit-for-purpose method development and validation for successful biomarker measurement. Pharm Res. 2006;23(2):312–28.
Article CAS PubMed Google Scholar
Findlay JW, Dillard RF. Appropriate calibration curve fitting in ligand binding assays. AAPS J. 2007;9(2):E260–7.
Article PubMed PubMed Central Google Scholar
Xiang Y, et al. A simple approach to determine a curve fitting model with a correct weighting function for calibration curves in quantitative ligand binding assays. AAPS J. 2018;20(3):45.
Article CAS PubMed Google Scholar
Piccoli SP, Brad Ackermann JMS, Allinson J, Arnold M, Amur S. Points to Consider Document: Scientific and Regulatory Considerations for the Analytical Validation of Assays Used in the Qualification of Biomarkers in Biological Matrices. C – Path; 2019.
Hammarström P, et al. Sequence-dependent denaturation energetics: a major determinant in amyloid disease diversity. Proc Natl Acad Sci. 2002;99(suppl_4):16427–32.
Article PubMed PubMed Central Google Scholar
Benson MD, et al. Inotersen treatment for patients with hereditary Transthyretin amyloidosis. N Engl J Med. 2018;379(1):22–31.
Article CAS PubMed PubMed Central Google Scholar
Jiang X, et al. An engineered transthyretin monomer that is nonamyloidogenic, unless it is partially denatured. Biochemistry. 2001;40(38):11442–52.
Article CAS PubMed Google Scholar
Ni YG, et al. Why is biomarker assay validation different from that of pharmacokinetic assays? AAPS J. 2025;27(6):147.
van der Vorm LN, et al. Toward Worldwide Hepcidin Assay Harmonization: Identification of a Commutable Secondary Reference Material. Clin Chem. 2016;62(7):993–1001.
Mollenhauer B, et al. Antibody-based methods for the measurement of α-synuclein concentration in human cerebrospinal fluid - method comparison and round robin study. J Neurochem. 2019;149(1):126–38.
Article CAS PubMed Google Scholar
Schoonenboom NS, et al. Differences and similarities between two frequently used assays for amyloid beta 42 in cerebrospinal fluid. Clin Chem. 2005;51(6):1057–60.
Article CAS PubMed Google Scholar
Arslan B, et al. Method comparison of plasma and CSF GFAP immunoassays across multiple platforms. Clinical Chemistry and Laboratory Medicine (CCLM). 2025. https://doi.org/10.1515/cclm-2025-0667.
Keller S, Purushothama S. Biomarker assays: is it time for a standalone regulatory guidance? Bioanalysis. 2018;10(23):1893–5.
Article CAS PubMed Google Scholar
Goodman J, et al. Update to the European Bioanalysis Forum recommendation on biomarkers assays; bringing context of use into practice. Bioanalysis. 2020;12(20):1427–37.
Article CAS PubMed Google Scholar
Health NIO. FDA-NIH Biomarker Working Group. BEST (Biomarkers, EndpointS, and other Tools) Resource. Bethesda (MD). 2016.
Krueger K, et al. High quality performance of novel immunoassays for the sensitive quantification of soluble PD-1, PD-L1 and PD-L2 in blood. Biomedicines. 2022;10(10):2405.
Article CAS PubMed PubMed Central Google Scholar
Soderstrom CI, et al. Comparison of four distinct detection platforms using multiple ligand binding assay formats. J Immunol Methods. 2011;371(1):106–13.
Article CAS PubMed Google Scholar
Lee S, et al. Development of a Highly Sensitive Neurofilament Light Chain Assay on an Automated Immunoassay Platform. Front Neurol. 2022;13:935382.
Comments (0)