The diflunisal trial: Update on study drug tolerance and disease progression

J. L. Berk, P. J. Dyck, L. Obici, S. R. Zeldenrust, Y. Sekijima, T. Yamashita, Y. Ando, S. I. Ikeda, P. Gorevic, G. Merlini, J. W. Kelly, M. Skinner, A. B. Bisbee, O. B. Suhr

Research output: Contribution to journalArticlepeer-review

14 Scopus citations


Familial amyloidotic polyneuropathy (FAP) is a lethal genetic disorder that affects the peripheral and autonomic nervous systems, heart, gastro-intestinal (GI) tract, and soft tissues. Disease progression is increasingly reported following liver transplantation, the only proven treatment for FAP. Small molecule thyroxine mimetics stabilize transthyretin, inhibiting FAP amyloid fibril formation under stringent in vitro conditions. We report on the progress of an international, randomized placebocontrolled study designed to determine the effect of diflunisal, a thyroxine mimetic, on neurologic disease progression in patients with active FAP. Our experience to date indicates diflunisal is well tolerated by this study cohort and that neurologic disease advances more rapidly in FAP than it does in diabetes mellitus.

Original languageEnglish (US)
Pages (from-to)196-197
Number of pages2
Issue numberSUPPL. 1
StatePublished - Jun 2011

ASJC Scopus subject areas

  • Internal Medicine


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