Tafamidis, a molecule first described by J. W. Kelly and co-workers in 2003, stabilizes the tetrameric protein transthyretin. When unstabilized transthyretin dissociates, misfolds, or aggregates, it causes amyloid deposition and can lead to the fatal diseases senile systemic amyloidosis, familial amyloid cardiomyopathy, and familial amyloid polyneuropathy. Tafamidis is well into Phase II and III clinical trials for treating these diseases.
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