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

J. L. Berk, Peter 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 journalArticle

12 Citations (Scopus)

Abstract

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
JournalAmyloid
Volume18
Issue numberSUPPL. 1
DOIs
StatePublished - Jun 2011

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Diflunisal
Drug Tolerance
Polyneuropathies
Disease Progression
Nervous System Diseases
Thyroxine
Autonomic Nervous System Diseases
Inborn Genetic Diseases
Prealbumin
Peripheral Nervous System
Amyloid
Liver Transplantation
Diabetes Mellitus
Cohort Studies

ASJC Scopus subject areas

  • Internal Medicine

Cite this

Berk, J. L., Dyck, P. J., Obici, L., Zeldenrust, S. R., Sekijima, Y., Yamashita, T., ... Suhr, O. B. (2011). The diflunisal trial: Update on study drug tolerance and disease progression. Amyloid, 18(SUPPL. 1), 196-197. https://doi.org/10.3109/13506129.2011.574354

The diflunisal trial : Update on study drug tolerance and disease progression. / Berk, J. L.; Dyck, Peter J; Obici, L.; Zeldenrust, S. R.; Sekijima, Y.; Yamashita, T.; Ando, Y.; Ikeda, S. I.; Gorevic, P.; Merlini, G.; Kelly, J. W.; Skinner, M.; Bisbee, A. B.; Suhr, O. B.

In: Amyloid, Vol. 18, No. SUPPL. 1, 06.2011, p. 196-197.

Research output: Contribution to journalArticle

Berk, JL, Dyck, PJ, Obici, L, Zeldenrust, SR, Sekijima, Y, Yamashita, T, Ando, Y, Ikeda, SI, Gorevic, P, Merlini, G, Kelly, JW, Skinner, M, Bisbee, AB & Suhr, OB 2011, 'The diflunisal trial: Update on study drug tolerance and disease progression', Amyloid, vol. 18, no. SUPPL. 1, pp. 196-197. https://doi.org/10.3109/13506129.2011.574354
Berk JL, Dyck PJ, Obici L, Zeldenrust SR, Sekijima Y, Yamashita T et al. The diflunisal trial: Update on study drug tolerance and disease progression. Amyloid. 2011 Jun;18(SUPPL. 1):196-197. https://doi.org/10.3109/13506129.2011.574354
Berk, J. L. ; Dyck, Peter J ; Obici, L. ; Zeldenrust, S. R. ; Sekijima, Y. ; Yamashita, T. ; Ando, Y. ; Ikeda, S. I. ; Gorevic, P. ; Merlini, G. ; Kelly, J. W. ; Skinner, M. ; Bisbee, A. B. ; Suhr, O. B. / The diflunisal trial : Update on study drug tolerance and disease progression. In: Amyloid. 2011 ; Vol. 18, No. SUPPL. 1. pp. 196-197.
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