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

Importance Familial amyloid polyneuropathy, a lethal genetic disease caused by aggregation
of variant transthyretin, induces progressive peripheral nerve deficits and disability. …

The nomenclature committee of the International Society of Amyloidosis (ISA) meets every
second year to discuss and formulate recommendations. The conclusions from the discussion …

The ISA Nomenclature Committee met electronically before and directly after the XVII ISA
International Symposium on Amyloidosis, which, unfortunately, had to be virtual in September …

Factors controlling the onset and progression of extracellular amyloid diseases remain
largely unknown. Central to disease etiology is the efficiency of the endoplasmic reticulum (ER) …

Background Transthyretin amyloidosis, also called ATTR amyloidosis, is associated with
accumulation of ATTR amyloid deposits in the heart and commonly manifests as progressive …

Friedreich ataxia (FRDA), the most common autosomal recessive neurodegenerative disease
among Europeans and people of European descent, is characterized by an early onset (…

The Nomenclature Committee of the International Society of Amyloidosis met at the XVIII
International Symposium on Amyloidosis in September and virtually in October 2022 with …

Small molecule-mediated protein stabilization inside or outside of the cell is a promising
strategy to treat protein misfolding/misassembly diseases. Herein we focus on the transthyretin (…

Transthyretin (ATTR) amyloidosis is a life-threatening, gain-of-toxic-function disease
characterised by extracellular deposition of amyloid fibrils composed of transthyretin (TTR). TTR …