Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial.

ABSTRACT:

Importance

Familial amyloid polyneuropathy, a lethal genetic disease caused by aggregation of variant transthyretin, induces progressive peripheral nerve deficits and disability. Diflunisal, a nonsteroidal anti-inflammatory agent, stabilizes transthyretin tetramers and prevents amyloid fibril formation in vitro.

Objective

To determine the effect of diflunisal on polyneuropathy progression in patients with familial amyloid polyneuropathy.

Design, setting, and participants

International randomized, double-blind, placebo-controlled study conducted among 130 patients with familial amyloid polyneuropathy exhibiting clinically detectable peripheral or autonomic neuropathy at amyloid centers in Sweden (Umeå), Italy (Pavia), Japan (Matsumoto and Kumamoto), England (London), a

SUBMITTER: Berk JL 

PROVIDER: S-EPMC4139164 | biostudies-literature | 2013 Dec

REPOSITORIES: biostudies-literature