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The Diflunisal Trial: Study accrual and drug tolerance

Academic Article
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Overview

authors

  • Berk, J. L.
  • Suhr, O. B.
  • Sekijima, Y.
  • Yamashita, T.
  • Heneghan, M.
  • Zeldenrust, S. R.
  • Ando, Y.
  • Ikeda, S.
  • Gorevic, P.
  • Merlini, G.
  • Kelly, Jeffery
  • Skinner, M.
  • Bisbee, A. B.
  • Dyck, P. J.
  • Obici, L.
  • Familial Amyloidosis Consortium

publication date

  • June 2012

journal

  • Amyloid-Journal of Protein Folding Disorders  Journal

abstract

  • Familial amyloidotic polyneuropathy (FAP) is a protein folding disorder that induces neuropathy and cardiomyopathy, leading to death within 7-15 years after onset of clinical disease. In vitro, small ligands binding the thyroid hormone docking site stabilize tetrameric transthyretin, inhibiting amyloid fibril formation. We undertook a randomized, placebo-controlled clinical trial to determine whether diflunisal, a well-known non-steroidal anti-inflammatory drug (NSAID) alters neurologic disease progression in FAP. We enrolled 130 subjects with wide age and FAP mutation representation. To date, few recognized complications of NSAIDs have occurred in the study cohort. Data collection will be completed by November 2012.

subject areas

  • Adult
  • Aged
  • Amyloid Neuropathies, Familial
  • Anti-Inflammatory Agents, Non-Steroidal
  • Diflunisal
  • Female
  • Humans
  • Male
  • Middle Aged
  • Young Adult
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Research

keywords

  • Clinical trials
  • FAP
  • diflunisal
  • neuropathy
  • transthyretin
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Identity

International Standard Serial Number (ISSN)

  • 1350-6129

Digital Object Identifier (DOI)

  • 10.3109/13506129.2012.678509

PubMed ID

  • 22551208
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Additional Document Info

start page

  • 37

end page

  • 38

volume

  • 19

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