Novel Drugs Targeting Transthyretin Amyloidosis
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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.
KeywordsTransthyretin Amyloid Amyloidosis Cardiac Cardiomyopathy Neuropathy Familial Senile Hereditary Pharmacologic Therapy Drugs
Compliance with Ethics Guidelines
Conflict of Interest
Mazen Hanna received a one-time consultant fee from Pfizer in April 2012 regarding input for the drug Tafamadis. He is also the local principal investigator of the Tafamadis Cardiomyopathy Trial and is a member of the Scientific Board for THAOS (Transthyretin Outcomes Survey), both sponsored by Pfizer.
Human and Animal Rights and Informed Consent
This article does not contain any studies with human or animal subjects performed by any of the authors.
Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance
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