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Background We previously showed the properties of interleukin-1b antagonist Anakinra on unmyelinated nerve fibers protection in a transgenic mouse model for human V30M transthyretin. In these studies, Anakinra decreased, among other markers, nerve levels of IL-1b, NFB and activated-caspase 3, associated with a decrease in TTRnon fibrillar deposition. In the present work, we compared the efficacy of other compounds under human therapeutical trials for Familial Amyloidotic Polyneuropathy (FAP) in the same animal model, having 5 months of age.

orphanet journal of rare diseases

Neuroprotection of Anakinra on peripheral nerve neurodegeneration in single and combination protocols with TTR siRNA in a transgenic mouse model for human V30M transthyretin