Transthyretin (TTR) is a tetrameric protein synthesized mostly by the liver and secreted into
the plasma. TTR molecules can misfold and form amyloid fibrils in the heart and peripheral …

Hereditary amyloidogenic transthyretin (ATTRv) amyloidosis with polyneuropathy (also
known as familial amyloid polyneuropathy) is a condition with adult onset caused by …

Background Patisiran, an investigational RNA interference therapeutic agent, specifically
inhibits hepatic synthesis of transthyretin. Methods In this phase 3 trial, we randomly …

Background Transthyretin amyloidosis is caused by the deposition of hepatocyte-derived
transthyretin amyloid in peripheral nerves and the heart. A therapeutic approach mediated …

Hereditary transthyretin-mediated amyloidosis is a rapidly progressive, heterogeneous
disease caused by the accumulation of misfolded transthyretin protein as amyloid fibrils at …

Hereditary transthyretin amyloidosis (hATTR) with polyneuropathy (formerly known as
Familial Amyloid Polyneuropathy) is a rare disease due to mutations in the gene encoding …

Background Patisiran is an investigational RNA interference (RNAi) therapeutic in
development for the treatment of hereditary ATTR (hATTR) amyloidosis, a progressive …

Transthyretin familial amyloid polyneuropathy (TTR-FAP) is a progressive, fatal, inherited
disorder first identified in Portugal and now recognized in all continents. Over the past …

Elevated pro-inflammatory biomarkers and cytokines are associated with morbidity and
mortality in heart failure (HF). Preclinical and clinical studies have shown multiple …

Amyloidoses represent a group of diseases characterized by the pathological accumulation
in the extracellular area of insoluble misfolded protein material called “amyloid”. The …