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 | |
| State | Published - Jun 2011 |
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ASJC Scopus subject areas
- Internal Medicine
Cite this
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 journal › Article
}
TY - JOUR
T1 - The diflunisal trial
T2 - Update on study drug tolerance and disease progression
AU - Berk, J. L.
AU - Dyck, Peter J
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.
PY - 2011/6
Y1 - 2011/6
N2 - 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.
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.
UR - http://www.scopus.com/inward/record.url?scp=79959948406&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=79959948406&partnerID=8YFLogxK
U2 - 10.3109/13506129.2011.574354
DO - 10.3109/13506129.2011.574354
M3 - Article
AN - SCOPUS:79959948406
VL - 18
SP - 196
EP - 197
JO - Amyloid
JF - Amyloid
SN - 1350-6129
IS - SUPPL. 1
ER -