Evaluation of tafamidis as first-line therapeutic agent for transthyretin familial amyloidotic polyneuropathy
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Almost 100 mutations in the human transthyretin (TTR) gene cause the autosomal dominant disorders of familial amyloidotic polyneuropathy (FAP) and familial amyloidotic cardiomyopathy. While these have been clinically classified as separate disorders, the perip... ...
