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  • Title: [Familial amyloid polyneuropathy: liver transplantation as first-line therapy].
    Author: Yamashita T.
    Journal: Brain Nerve; 2014 Jul; 66(7):763-71. PubMed ID: 24998821.
    Abstract:
    Liver transplantation is the only potentially curative treatment currently available for transthyretin familial amyloid polyneuropathy (TTR-FAP) since transplantation results in the disappearance of amyloidogenic TTR, synthesized by the original liver, from plasma. An absolute risk reduction of 66.3%, class III evidence, and grade B recommendation demonstrate that liver transplantation prolongs survival in patients with FAP Val30Met. Effect of new therapeutic drugs should be compared with that of liver transplantation, and outcome improvement of therapy for FAP is expected.
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