Targeted Therapeutics for Transthyretin Cardiac Amyloidosis.

Abstract

Despite the availability of proven treatments for some patients with heart failure (HF), many patients—particularly those with HF and preserved ejection fraction (HFpEF)—remain difficult to treat, resulting in persistently high morbidity and mortality in the majority of HF patients. The lack of effective treatments, disappointing results of many HF randomized clinical trials (RCT), and variable treatment responses even for proven therapies are all possible reasons for the poor prognosis of HF patients. In the context of this backdrop, it is not surprising that there has been growing interest and enthusiasm for “precision medicine” in order to improve outcomes for patients who suffer from the HF syndrome.^,