Novel drugs targeting transthyretin amyloidosis.
Transthyretin amyloidosis (ATTR) is either a hereditary disease related to a mutation in the transthyretin gene that leads to neuropathy and/or cardiomyopathy or an acquired disease of the elderly that leads to restrictive cardiomyopathy. The prevalence of this disease is higher than once thought and awareness is likely to increase amongst physicians and in particular cardiologists. Until recently there have been no treatment options for this disease except to treat the heart failure with diuretics and the neuropathy symptomatically. However, there are several emerging pharmacologic therapies designed to slow or stop the progression of ATTR. This article reviews novel therapeutic drugs that work at different points in the pathogenesis of this disease attempting to change its natural history and improve outcomes.
Section of Heart Failure and Cardiac Transplantation, Cleveland Clinic, 9500 Euclid Avenue, Desk J3-4, Cleveland, OH, 44195, USA, hannam@ccf.org.
Mazen
M
Hanna
D028227:Amyloid Neuropathies, Familial / Q000150:complications / Q000188:drug therapy* / Q000235:genetics; D002313:Cardiomyopathy, Restrictive / Q000209:etiology / Q000517:prevention & control; D006801:Humans; D058990:Molecular Targeted Therapy / Q000379:methods*; D009154:Mutation; D011228:Prealbumin / Q000235:genetics / Q000502:physiology
D011228:Prealbumin