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Familial amyloidotic polyneuropathy: current and emerging treatment options for transthyretin-mediated amyloidosis

Transthyretin familial amyloid polyneuropathy (TTR-FAP) is a fatal clinical disorder characterized by extracellular deposition of abnormal fibrils derived from misfolded, normally soluble transthyretin (TTR) molecules. The disease is most commonly caused by a point mutation within the TTR gene inher...

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Библиографические подробности
Главный автор: Hund, Ernst
Формат: Artigo
Язык:Inglês
Опубликовано: Dove Medical Press 2012
Предметы:
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC3681191/
https://ncbi.nlm.nih.gov/pubmed/23776379
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/TACG.S19903
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