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[Newly developed drug therapies for familial amyloid polyneuropathy: diflunisal and tafamidis].

Authors
Type
Published Article
Journal
Brain and nerve = Shinkei kenkyū no shinpo
Publication Date
Volume
66
Issue
7
Pages
773–781
Identifiers
PMID: 24998822
Source
Medline
License
Unknown

Abstract

Familial amyloid polyneuropathy (FAP) is an autosomal dominant genetic disorder with systemic deposition of amyloid fibrils, and is characterized by progressive sensory, motor, and autonomic polyneuropathy. FAP was considered a rare endemic disease; however, its worldwide incidence is much higher than previously recognized. Until recently, liver transplantation was the only effective treatment for FAP. However, liver transplantation has a number of limitations, including a shortage of donors and a requirement for surgery for both the recipient and living donor. Furthermore, a large number of the patients are not good transplant candidates because of their age and/or advanced disease status. Recently, the clinical effects of two transthyretin tetramer stabilizers, diflunisal and tafamidis, were demonstrated in randomized clinical trials, and tafamidis was approved for the treatment of FAP in European countries in 2011 and Japan in 2013.

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