• 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, and Familial Amyloidosis Consortium.
• Department of Medicine & Data Coordinating Center, Boston University Medical Center, Boston, MA 02118, USA. jberk@bu.edu
• Amyloid. 2012 Jun 1;19 Suppl 1:37-8.

AbstractFamilial 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.

17 keywords...

hide…