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Amyloid
The Journal of Protein Folding Disorders
Volume 23, 2016 - Issue 3
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Original Article

Early intervention with tafamidis provides long-term (5.5-year) delay of neurologic progression in transthyretin hereditary amyloid polyneuropathy

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Pages 178-183 | Received 27 Jan 2016, Accepted 26 Jun 2016, Published online: 05 Aug 2016

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Catarina Falcão de Campos & Isabel Conceição. (2023) Updated Evaluation of the Safety, Efficacy and Tolerability of Tafamidis in the Treatment of Hereditary Transthyretin Amyloid Polyneuropathy. Drug, Healthcare and Patient Safety 15, pages 51-62.
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Teresa Coelho, Isabel Conceição, Márcia Waddington-Cruz, Denis Keohane, Marla B. Sultan, Doug Chapman & Leslie Amass. (2022) A natural history analysis of asymptomatic TTR gene carriers as they develop symptomatic transthyretin amyloidosis in the Transthyretin Amyloidosis Outcomes Survey (THAOS). Amyloid 29:4, pages 228-236.
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Arianna Burton, Adam Castaño, Marianna Bruno, Steve Riley, Jennifer Schumacher, Marla B Sultan, Sandi See Tai, Daniel P Judge, Jignesh K Patel & Jeffery W Kelly. (2021) Drug Discovery and Development in Rare Diseases: Taking a Closer Look at the Tafamidis Story. Drug Design, Development and Therapy 15, pages 1225-1243.
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Matthias N. Ungerer, Ernst Hund, Jan C. Purrucker, Laura Huber, Christoph Kimmich, Fabian aus dem Siepen, Selina Hein, Arnt V. Kristen, Katrin Hinderhofer, Jennifer Kollmer, Stefan Schönland, Ute Hegenbart & Markus Weiler. (2021) Real-world outcomes in non-endemic hereditary transthyretin amyloidosis with polyneuropathy: a 20-year German single-referral centre experience. Amyloid 28:2, pages 91-99.
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Ola Akinboboye, Keyur Shah, Alberta L. Warner, Thibaud Damy, Herman A. Taylor, Jared Gollob, Christine Powell, Verena Karsten, John Vest & Mathew S. Maurer. (2020) DISCOVERY: prevalence of transthyretin (TTR) mutations in a US-centric patient population suspected of having cardiac amyloidosis. Amyloid 27:4, pages 223-230.
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Maria C. Azevedo Coutinho, Nuno Cortez-Dias, Guilhermina Cantinho, Susana Gonçalves, Miguel Nobre Menezes, Tatiana Guimarães, Gustavo Lima da Silva, Ana Rita Francisco, João Agostinho, Laura Santos, Isabel Conceição & Fausto J. Pinto. (2020) The sensitivity of DPD scintigraphy to detect transthyretin cardiac amyloidosis in V30M mutation depends on the phenotypic expression of the disease. Amyloid 27:3, pages 174-183.
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Elissa Driggin, Stephen Helmke, Jeffeny De Los Santos, Sergio Teruya, Samantha Guadalupe, Jeff Goldsmith & Mathew S. Maurer. (2020) Markers of nutritional status and inflammation in transthyretin cardiac amyloidosis: association with outcomes and the clinical phenotype. Amyloid 27:2, pages 73-80.
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Isabel Conceição, Teresa Coelho, Claudio Rapezzi, Yeşim Parman, Laura Obici, Lucía Galán & Antoine Rousseau. (2019) Assessment of patients with hereditary transthyretin amyloidosis – understanding the impact of management and disease progression. Amyloid 26:3, pages 103-111.
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Jonah Rubin, Julissa Alvarez, Sergio Teruya, Adam Castano, Ronald A. Lehman, Mark Weidenbaum, Jeffrey A. Geller, Stephen Helmke & Mathew S. Maurer. (2017) Hip and knee arthroplasty are common among patients with transthyretin cardiac amyloidosis, occurring years before cardiac amyloid diagnosis: can we identify affected patients earlier?. Amyloid 24:4, pages 224-228.
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Hirofumi Jono & Yukio Ando. (2017) Towards targets and treatments in transthyretin amyloidosis. Expert Opinion on Orphan Drugs 5:9, pages 691-699.
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Fabio A. Barroso, Daniel P. Judge, Ben Ebede, Huihua Li, Michelle Stewart, Leslie Amass & Marla B. Sultan. (2017) Long-term safety and efficacy of tafamidis for the treatment of hereditary transthyretin amyloid polyneuropathy: results up to 6 years. Amyloid 24:3, pages 194-204.
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Denis Keohane, Jeffrey Schwartz, Balarama Gundapaneni, Michelle Stewart & Leslie Amass. (2017) Tafamidis delays disease progression in patients with early stage transthyretin familial amyloid polyneuropathy: additional supportive analyses from the pivotal trial. Amyloid 24:1, pages 30-36.
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