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Article

Patisiran, an RNAi Therapeutic for the Treatment of Hereditary Transthyretin-Mediated Amyloidosis

27 Nov 2018

Abstract excerpt

Hereditary transthyretin-mediated amyloidosis is a rapidly progressive, heterogeneous disease caused by the accumulation of misfolded transthyretin protein as amyloid fibrils at multiple sites, and is characterized by peripheral sensorimotor neuropathy, autonomic neuropathy and/or cardiomyopathy. Current treatment options have limited efficacy and often do not prevent disease progression. Patisiran is a novel RNA...

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Share to Amyloidosis: Diagnosis, Treatment, Outcomes
Share to Cellular transport and secretion
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PMID: 30480471
DOI: 10.2217/nmt-2018-0033

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