Clinical Trials and Research

INTRODUCTIONAcquired transthyretin amyloidosis (ATTR) represents a rare but recognized complication in recipients of domino liver transplants (DLT) from donors…

The cross-border transfer of machine learning models based on the International Classification of Diseases, 10th revision (ICD-10) for the identification of rare…

AbstractBackgroundTransthyretin amyloid cardiomyopathy (ATTR-CM) causes heart failure with substantial morbidity, mortality, and healthcare utilization. ATTRibute-…

The global CARES phase 3 clinical programme has shown that treatment with anselamimab, a potential first-in-class anti-fibril therapy, resulted in nominally…

AbstractIntroductionBlack individuals have significantly lower serum N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels than White individuals in the…

AbstractBackgroundPlasma transthyretin (TTR) is routinely quantified in clinical laboratories, yet the structural heterogeneity of circulating TTR and its potential…

Monitoring success in AL amyloidosis is a long-term journey. Managing the disease involves keeping a close eye on two different timelines: the speed of the “factory…

AbstractWild-type transthyretin amyloid (ATTRwt) amyloidosis is a systemic, progressive disease that often affects older adults and is characterized by ATTRwt…

Cardiac amyloidosis is an increasingly recognized infiltrative cardiomyopathy characterized by extracellular deposition of misfolded amyloid fibrils within the…

AbstractAims: Eplontersen is an antisense oligonucleotide that suppresses hepatic production of circulating transthyretin (TTR) by targeting TTR mRNA. We present…