SOM0226, a repositioned compound for the treatment of TTR amyloidosis
نویسندگان
چکیده
Background Transthyretin amyloidosis is a protein aggregation disorder caused by deposition of transthyretin, a tetrameric plasma protein in charge of the transport of thyroxin and retinol in plasma and cerebrospinal fluid. Kinetic stabilization of the TTR tetramer has proved to be a valid therapeutic strategy to prevent the release of unstable TTR monomers and their aggregation into oligomers and amyloid fibrils. SOM0226 is tolcapone, a repositioned compound with a newly identified activity as a potent TTR stabilizer and TTR fibril disruptor.
منابع مشابه
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