Hereditary transthyretin-mediated (hATTR) amyloidosis is a progressive, life-threatening disease. The majority of patients develop a mixed phenotype of polyneuropathy and cardiomyopathy. Patisiran is approved in certain countries globally for the… Click to show full abstract
Hereditary transthyretin-mediated (hATTR) amyloidosis is a progressive, life-threatening disease. The majority of patients develop a mixed phenotype of polyneuropathy and cardiomyopathy. Patisiran is approved in certain countries globally for the treatment of hATTR amyloidosis with polyneuropathy.
               
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