The aggregation of proteins into amyloid fibrils and their deposition into plaques and
intracellular inclusions is the hallmark of amyloid disease. The accumulation and deposition …

Often considered a rare disease, cardiac amyloidosis is increasingly recognized by
practicing clinicians. The increased rate of diagnosis is in part due the aging of the …

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

The recent approval of three drugs for the treatment of amyloid transthyretin (ATTR)
amyloidosis, both hereditary and wild-type, has opened a new era in the care of these …

Objectives: To evaluate the efficacy and safety of 18 months of tafamidis treatment in
patients with early-stage V30M transthyretin familial amyloid polyneuropathy (TTR-FAP) …

The transthyretin amyloidoses (ATTR) are invariably fatal diseases characterized by
progressive neuropathy and/or cardiomyopathy. ATTR are caused by aggregation of …

Peptides or proteins convert under some conditions from their soluble forms into highly
ordered fibrillar aggregates. Such transitions can give rise to pathological conditions ranging …

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

The systemic amyloidoses are a group of complex diseases caused by tissue deposition of
misfolded proteins that results in progressive organ damage. The most common type …

Transthyretin (TTR) is a tetrameric protein synthesized mostly by the liver. As a result of gene
mutations or as an ageing-related phenomenon, TTR molecules may misfold and deposit in …