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Description: Tafamidis (also known as Fx-1006 or PF-06291826), a potent and selective transthyretin (TTR) stabilizer, is a drug for the amelioration of transthyretin-related hereditary amyloidosis (also familial amyloid polyneuropathy, or FAP), a rare but deadly neurodegenerative disease. It shows comparable potency and efficacy to the mutumant homotetramers V30M-TTR, V122I-TTR and wild type WT-TTR, with EC50s of 2.7-3.2 uM. Tafamidis inhibits amyloidogenesis. The drug was approved by the European Medicines Agency in November 2011 and by the Japanese Pharmaceuticals and Medical Devices Agency in September 2013. Tafamidis functions by kinetic stabilization of the correctly folded tetrameric form of the transthyretin (TTR) protein.
References: Proc Natl Acad Sci U S A, 2012. 109(24): p. 9629-34.
Related CAS: 951395-08-7 (meglumine salt)
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