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Amyloidosis is a protein folding disease that causes organ injuries and even death. In humans, 42 proteins are now known to cause amyloidosis. Some proteins become…

AL amyloidosis is caused by the excessive production of nonfunctional immunoglobulins, leading to the formation of amyloid fibrils that damage vital organs,…

– Family Health History Road Trip Program Aims to Inspire Conversations About Health History and Empower Families to Take Control of Their Health ––…

Full Article hATTR Holistic Care Consensus Reccomendations BMJexternal link, opens in a new tabAbstractBackground Hereditary transthyretin-mediated amyloidosis is a…

About the episodeIn this video, Prof Philip Hawkins (University College London, London, UK), addresses some important key questions surrounding amyloidosis, a…

FDA is developing a series of four methodological patient-focused drug development (PFDD) guidance documents to address, in a stepwise manner, how stakeholders can…

BackgroundAmyloidosis represents a rare yet heterogeneous multi-system disorder associated with a grave prognosis and an enormous psycho-emotional strain on…

AimsHELIOS-A was a Phase 3, open-label study of vutrisiran, an RNA interference therapeutic, in patients with hereditary transthyretin (ATTRv) amyloidosis with…

BackgroundTransthyretin cardiac amyloidosis (ATTR‐CA) is a progressive and ultimately fatal cardiomyopathy. Biomarkers reflecting multiorgan dysfunction are of…

KEY POINTSTransthyretin amyloidosis typically presents with entrapment neuropathy, polyneuropathy or heart failure; some patients may experience autonomic…