Potent and selective transthyretin (TTR) kinetic stabilizer. It binds selectively and with negative cooperativity to the two normally unoccupied thyroxine binding sites of the TTR tetramer (Kd = ~2 and ~200 nM). Stabilizes wild-type and mutant TTR under varying conditions in vitro (EC50=2.7-3.2...
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Potent and selective transthyretin (TTR) kinetic stabilizer. It binds selectively and with negative cooperativity to the two normally unoccupied thyroxine binding sites of the TTR tetramer (Kd = ~2 and ~200 nM). Stabilizes wild-type and mutant TTR under varying conditions in vitro (EC50=2.7-3.2 μM). Clinically useful for the treatment of familial amyloid polyneuropathy and TTR-related cardiomyopathy
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