Transthyretin amyloidosis is a fatal disorder that is characterized primarily by progressive
neuropathy and cardiomyopathy. It occurs in both a mutant form (with autosomal dominant …

Hereditary transthyretin amyloidosis (ATTRv) is a severe, adult-onset autosomal dominant
inherited systemic disease predominantly affecting the peripheral and autonomic nervous …

The understanding of why and how proteins misfold and aggregate into amyloid fibrils has
increased considerably during recent years. Central to amyloid formation is an increase in …

The abnormal aggregation of tau protein into paired helical filaments (PHFs) is one of the
hallmarks of Alzheimer's disease. Aggregation takes place in the cytoplasm and could …

We designed a phase II, open-label study to evaluate the efficacy, tolerability, safety, and
pharmacokinetics of orally doxycycline (100 mg BID) and tauroursodeoxycholic acid …

Transthyretin (TTR) is a plasma homotetrameric protein implicated in fatal systemic
amyloidoses. TTR tetramer dissociation precedes pathological TTR aggregation. Native …

Cardiac involvement in patients with amyloidosis is common. It produces significant clinical
symptoms in about 40% of patients with AL amyloidosis. A significant proportion of patients …

Knowledge about the systemic amyloidoses has increased considerably during the last few
years. This group of diseases is characterized by great biochemical variability, including at …

Light chain (AL) amyloidosis is caused by a usually small plasma-cell clone that is able to
produce the amyloidogenic light chains. They are able to misfold and aggregate, deposit in …

Abstract Familial Amyloidotic Polyneuropathy (FAP) is a disorder characterized by the
extracellular deposition of fibrillar Transthyretin (TTR) amyloid, with a special involvement of …