References
- Hund E. Familial amyloidotic polyneuropathy: current and emerging treatment options for transthyretin-mediated amyloidosis. Appl Clin Genet. 2012;5:37–41.
- Planté-Bordeneuve V, Said G. Familial amyloid polyneuropathy. Lancet Neurol. 2011;10:1086–1097.
- Adams D, Cauquil C, Labeyrie C. Familial amyloid polyneuropathy. Curr Opin Neurol. 2017;30:481–489.
- Adams D, Suhr OB, Hund E, et al. European Network for TTR-FAP (ATTReuNET). First European consensus for diagnosis, management, and treatment of transthyretin familial amyloid polyneuropathy. Curr Opin Neurol. 2016;29:S14–S26.
- Parman Y, Adams D, Obici L, et al. Sixty years of transthyretin familial amyloid polyneuropathy (TTR-FAP) in Europe: where are we now? A European network approach to defining the epidemiology and management patterns for TTR-FAP. Curr Opin Neurol. 2016;29:S3–S13.
- Plante-Bordeneuve V. Update in the diagnosis and management of transthyretin familial amyloid polyneuropathy. J Neurol. 2014;261:1227–1233.
- Herlenius G, Wilczek HE, Larsson M, et al. Ten years of international experience with liver transplantation for familial amyloidotic polyneuropathy: results from the Familial Amyloidotic Polyneuropathy World Transplant Registry. Transplantation. 2004;77:64–71.
- Carvalho A, Rocha A, Lobato L. Liver transplantation in transthyretin amyloidosis: issues and challenges. Liver Transpl. 2015;21:282–292.
- Liepnieks JJ, Zhang LQ, Benson MD. Progression of transthyretin amyloid neuropathy after liver transplantation. Neurology. 2010;75:324–327.
- Oshima T, Kawahara S, Ueda M, et al. Changes in pathological and biochemical findings of systemic tissue sites in familial amyloid polyneuropathy more than 10 years after liver transplantation. J Neurol Neurosurg Psychiatry. 2014;85:740–746.
- Berk JL, Suhr OB, Obici L, et al. Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial. JAMA. 2013;310:2658–2667.
- Coelho T, Maia LF, Martins da Silva A, et al. Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial. Neurology. 2012;79:785–792.
- Coelho T, Adams D, Silva A, et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013;369:819–829.
- Maurer MS, Heitner S, Drachman B, et al. Inotersen improves quality of life in patients with hereditary transthyretin amyloidosis with polyneuropathy and cardiomyopathy: results of the phase 3 study NEURO-TTR. J Am Coll Cardiol. 2018;71:A658.
- Planté-Bordeneuve V, Gorram F, Salhi H, et al. Long-term treatment of transthyretin familial amyloid polyneuropathy with tafamidis: a clinical and neurophysiological study. J Neurol. 2017;264:268–276.
- Ueda M, Ando Y. Recent advances in transthyretin amyloidosis therapy. Transl Neurodegener. 2014;3:19.
- Adams D, Cauquil C, Labeyrie C, et al. TTR kinetic stabilizers and TTR gene silencing: a new era in therapy for familial amyloidotic polyneuropathies. Expert Opin Pharmacother. 2016;17:791–802.
- Li YY, Jones SJ. Drug repositioning for personalized medicine. Genome Med. 2012;4:27.
- Cacabelos R. Parkinson's disease: from pathogenesis to pharmacogenomics. IJMS. 2017;18:551.
- Stowe R, Ives N, Clarke CE, et al. Meta-analysis of the comparative efficacy and safety of adjuvant treatment to levodopa in later Parkinson's disease. Mov Disord. 2011;26:587–598.
- Sant'Anna R, Gallego P, Robinson LZ, et al. Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity. Nat Commun. 2016;7:10787.
- Medical Research Council of the UK. Aids to the investigation of peripheral nerve injuries, Memorandum No.45. London (UK): Pendragon House; 1976. p. 1–2.
- Breiner A, Barnett C, Bril V. INCAT disability score: a critical analysis of its measurement properties. Muscle Nerve. 2014;50:164–169.
- https://www.ema.europa.eu/medicines/human/EPAR/tasmar. Updated 2018 September 5; Cited 2019 April 24.
- Kullak-Ublick GA, Andrade RJ, Merz M, et al. Drug-induced liver injury: recent advances in diagnosis and risk assessment. Gut. 2017;66:1154–1164.
- Dingemanse J, Jorga KM, Schmitt M, et al. Integrated pharmacokinetics and pharmacodynamics of the novel catechol-O-methyltransferase inhibitor tolcapone during first administration to humans. Clin Pharmacol Ther. 1995;57:508–517.
- Dingemanse J, Jorga K, Zürcher G, et al. Pharmacokinetic-pharmacodynamic interaction between the COMT inhibitor tolcapone and single-dose levodopa. Br J Clin Pharmacol. 1995;40:253–262.
- Dingemanse J, Jorga K, Zürcher G, et al. Multiple-dose clinical pharmacology of the catechol-O-methyl-transferase inhibitor tolcapone in elderly subjects. Eur J Clin Pharmacol. 1996;50:47–55.
- Jorga K, Fotteler B, Banken L, et al. Population pharmacokinetics of tolcapone in parkinsonian patients in dose finding studies. Br J Clin Pharmacol. 2000;49:39–48.
- Blirup-Jensen S. Protein standardization III: method optimization basic principles for quantitative determination of human serum proteins on automated instruments based on turbidimetry or nephelometry. Clin Chem Lab Med. 2001;39:1098–1109.
- Bulawa CE, Connelly S, Devit M, et al. Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade. Proc Natl Acad Sci U S A. 2012;109:9629–9634.
- https://www.ema.europa.eu/documents/scientific-guideline/guideline-bioanalytical-method-validation_en.pdf. Updated 2011 July 21. Cited 2019 April 24.
- Kerschen P, Planté-Bordeneuve V. Current and future treatment approaches in transthyretin familial amyloid polyneuropathy. Curr Treat Options Neurol. 2016;18:53.
- Verona G, Mangione PP, Raimondi S, et al. Inhibition of the mechano-enzymatic amyloidogenesis of transthyretin: role of ligand affinity, binding cooperativity and occupancy of the inner channel. Sci Rep. 2017;7:182.