The Diflunisal Trial: Study accrual and drug tolerance

John L. Berk, Ole B. Suhr, Yoshiki Sekijima, Taro Yamashita, Michael Heneghan, Steven R. Zeldenrust, Yukio Ando, Shu Ichi Ikeda, Peter Gorevic, Giampaolo Merlini, Jeffrey W. Kelly, Martha Skinner, Alice B. Bisbee, Peter J. Dyck, Laura Obici

Research output: Contribution to journalArticle

Abstract

Familial amyloidotic polyneuropathy (FAP) is a protein folding disorder that induces neuropathy and cardiomyopathy, leading to death within 715 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.

Original languageEnglish
Pages (from-to)37-38
Number of pages2
JournalAmyloid
Volume19
Issue numberSUPPL. 1
DOIs
Publication statusPublished - Jun 2012

Keywords

  • Clinical trials
  • Diflunisal
  • FAP
  • Neuropathy
  • transthyretin

ASJC Scopus subject areas

  • Internal Medicine

Fingerprint Dive into the research topics of 'The Diflunisal Trial: Study accrual and drug tolerance'. Together they form a unique fingerprint.

  • Cite this

    Berk, J. L., Suhr, O. B., Sekijima, Y., Yamashita, T., Heneghan, M., Zeldenrust, S. R., Ando, Y., Ikeda, S. I., Gorevic, P., Merlini, G., Kelly, J. W., Skinner, M., Bisbee, A. B., Dyck, P. J., & Obici, L. (2012). The Diflunisal Trial: Study accrual and drug tolerance. Amyloid, 19(SUPPL. 1), 37-38. https://doi.org/10.3109/13506129.2012.678509