Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
8537006 | Pharmacology & Therapeutics | 2017 | 41 Pages |
Abstract
Amyloidosis refers to a range of protein misfolding disorders that can cause organ dysfunction through progressive fibril deposition. Cardiac involvement often leads to significant morbidity and mortality and increasingly has been recognized as an important cause of heart failure. The two main forms of cardiac amyloidosis, light chain (AL) and transthyretin (ATTR) amyloidosis, have distinct mechanisms of pathogenesis. Recent insights have led to the development of novel pharmacotherapies with the potential to significantly impact each disease. This review will summarize the preclinical and clinical data for these emerging treatments for AL and ATTR amyloidosis.
Keywords
NT-proBNPtransthyretinFDAcTNTATTRRCTTUDCAEGCGN-terminal pro-B-type natriuretic peptidesmall-interfering RNAsiRNARandomized controlled trialAmyloidosisimmunoglobulin light chain amyloidosisTauroursodeoxycholic acidTransthyretin amyloidosiscardiac troponin TSerum amyloid P componentTherapylight chainFood and Drug AdministrationSAPNeuropathyCardiomyopathy
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Authors
Kevin M. Alexander, Avinainder Singh, Rodney H. Falk,