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FAP Neuropathy and Emerging Treatments.
- Source :
- Current Neurology & Neuroscience Reports; Mar2014, Vol. 14 Issue 3, p1-12, 12p
- Publication Year :
- 2014
-
Abstract
- Transthyretin familial amyloid polyneuropathy (TTR-FAP) classically presents as a length dependent small fiber polyneuropathy in endemic countries like Portugal. In nonendemic countries, it may mimic a variety of chronic polyneuropathies, with several phenotypes: ataxic, upper limb onset neuropathy, or motor. In these cases, there is usually a late onset and no positive family history. TTR gene sequencing appears the most pertinent first-line test for diagnosis. Cardiac involvement of various severities is common in FAP. Liver transplantation remains the standard antiamyloid therapy with better results in Val30Met TTR-FAP of early onset. Antiamyloid medication has been developed. (1) TTR stabilizers: Tafamidis was the first drug approved in Europe in stage 1 (walking unaided) TTR-FAP to slow progression of the disease; diflunisal has been assessed in a phase 3 clinical trial; (2) TTR gene silencing is a new strategy to inhibit production of both mutant and nonmutant TTR with antisense oligonucleotides or SiRNA (2 ongoing phase 3 clinical trials). [ABSTRACT FROM AUTHOR]
Details
- Language :
- English
- ISSN :
- 15284042
- Volume :
- 14
- Issue :
- 3
- Database :
- Complementary Index
- Journal :
- Current Neurology & Neuroscience Reports
- Publication Type :
- Academic Journal
- Accession number :
- 94799771
- Full Text :
- https://doi.org/10.1007/s11910-013-0435-3