Clinical Trials and Research

Attruby (acoramidis), an approved therapy for transthyretin amyloid cardiomyopathy (ATTR-CM), may help preserve kidney function in adults with the rare heart…

AbstractAccurate amyloid typing in cardiac amyloidosis is clinically important for determining treatment strategies. A 73-year-old man underwent a renal biopsy due…

HighlightsSubtle age-related ATTR-CM can complicate preexisting other HF etiologies.Combined cardiomyopathies can present with atypical clinical/imaging…

SummaryOnce rapidly fatal, neglected, and orphan diseases without approved therapeutic options, systemic amyloidoses are now highly treatable. Basic scientific…

Long-term treatment with Amvuttra (vutrisiran) was found to be safe and to sustain disease stability in people with hereditary transthyretin amyloidosis with…

Long-term treatment with Amvuttra (vutrisiran) was found to be safe and to sustain disease stability in people with hereditary transthyretin amyloidosis with…

AbstractBACKGROUND:Acoramidis achieves near-complete (≥90%) transthyretin stabilization and is approved to reduce cardiovascular-related hospitalization in…

Study OverviewBrief SummaryAL (or light chain) amyloidosis begins in the bone marrow where abnormal proteins misfold and create free light chains that cannot be…

AbstractThe most common hereditary transthyretin (ATTRv) amyloidosis variant in the United States, Val122Ile (p.Val142Ile), is predominantly detected in populations…

Collecting and analyzing wrist tissue for the presence of hallmark toxic protein clumps may not be sufficient as a stand-alone strategy for early detection of…