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

Tissue deposition of protein fibrils causes a group of rare diseases called systemic
amyloidoses. This Seminar focuses on changes in their epidemiology, the current approach …

Background Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive,
often fatal disease. Nexiguran ziclumeran (nex-z) is an investigational therapy based on …

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

The aggregation of specific proteins is hypothesized to underlie several degenerative
diseases, which are collectively known as amyloid disorders. However, the mechanistic …

Purpose To identify the criteria for hematologic and cardiac response to treatment in
immunoglobulin light chain (AL) amyloidosis based on survival analysis of a large patient …

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

Background Transthyretin-mediated amyloidosis is an inherited, progressively debilitating
disease caused by mutations in the transthyretin gene. This study evaluated the safety …

We undertook this study to develop uniformly accepted criteria for the definition of organ
involvement and response for patients on treatment protocols for immunoglobulin light …

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 …