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

doi:10.3109/13506129.2011.574354

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

Neurology

Research output: Contribution to journal › Article › peer-review

14 Scopus citations

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 language	English (US)
Pages (from-to)	196-197
Number of pages	2
Journal	Amyloid
Volume	18
Issue number	SUPPL. 1
DOIs	https://doi.org/10.3109/13506129.2011.574354
State	Published - Jun 2011

ASJC Scopus subject areas

Internal Medicine

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title = "The diflunisal trial: Update on study drug tolerance and disease progression",

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.",

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

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AU - Obici, L.

AU - Zeldenrust, S. R.

AU - Sekijima, Y.

AU - Yamashita, T.

AU - Ando, Y.

AU - Ikeda, S. I.

AU - Gorevic, P.

AU - Merlini, G.

AU - Kelly, J. W.

AU - Skinner, M.

AU - Bisbee, A. B.

AU - Suhr, O. B.

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AB - 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.

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

Abstract

ASJC Scopus subject areas

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