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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| Format: | Online |
| Language: | English |
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Dove Medical Press
2012
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| Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3681191/ |