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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…

Hereditary transthyretinexternal link, opens in a new tab amyloidosis is autosomal dominantexternal link, opens in a new tab and results from mutations in the…

ObjectivesThis study has been conducted to investigate the non-invasive diagnostic journey of patients with a transthyretin amyloid cardiomyopathy (aTTR-CM) in…

Cardiac amyloidosis is a type of amyloidosis that deserves special attention as organ involvement significantly worsens the prognosis. Cardiac amyloidosis can be…

Wild-type transthyretin amyloid cardiomyopathy (ATTRwt-CM) is an underrecognized cause of heart failure due to misfolded wild-type transthyretin (TTRwt) myocardial…

BackgroundStakeholders increasingly expect research and care delivery to be guided by and to optimize patient experiences. However, standardized tools to engage…