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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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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Dove Medical Press
2012
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3681191/ https://www.ncbi.nlm.nih.gov/pubmed/23776379 http://dx.doi.org/10.2147/TACG.S19903 |