RNA interference in late-stage hereditary transthyretin amyloidosis: a clinicopathological study
Author:
Funder
Japan Society for the Promotion of Science
Japan Agency for Medical Research and Development
Publisher
Springer Science and Business Media LLC
Subject
Neurology (clinical),Neurology
Link
https://link.springer.com/content/pdf/10.1007/s00415-023-11754-7.pdf
Reference15 articles.
1. Ando Y, Adams D, Benson MD et al (2022) Guidelines and new directions in the therapy and monitoring of ATTRv amyloidosis. Amyloid 29:143–155
2. Ruberg FL, Berk JL (2012) Transthyretin (TTR) cardiac amyloidosis. Circulation 126:1286–1300
3. Coelho T, Maia LF, da Silva AM et al (2013) Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy. J Neurol 260:2802–2814
4. Adams D, Gonzalez-Duarte A, O’Riordan WD et al (2018) Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med 379:11–21
5. Adams D, Polydefkis M, González-Duarte A et al (2021) Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study. Lancet Neurol 20:49–59
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