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TTR-FAP: A rare disease that causes neurodegeneration1,2
Transthyretin familial amyloid polyneuropathy (TTR-FAP) is a rare, progressive, irreversible, and fatal neurodegenerative disease primarily caused by a genetic mutation of TTR.1,2
In TTR-FAP, mutations in TTR lead to the formation of amyloid fibrils that deposit in peripheral and autonomic nerve tissues, resulting in neurodegeneration and decline of neurologic function.5,10 As the disease progresses, TTR-FAP eventually becomes fatal.11,12
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