Hereditary ATTR Amyloidosis (hATTR)
Patients may present with peripheral and/or autonomic neuropathy, or symptoms associated with cardiomyopathy. Many present with a mixed phenotype, which may include sensory, motor, autonomic or cardiac symptoms. In addition, accumulation of amyloid in the eyes, kidneys, thyroid and adrenal glands and blood vessels can lead to a diverse range of clinical manifestations of the disease. Diagnosing hereditary ATTR amyloidosis therefore often requires multidisciplinary collaboration.
The liver is responsible for the production of TTR and therefore liver transplantation has long been used in the treatment of hereditary ATTR amyloidosis. More recently, the development of drugs that stabilise TTR tetramers as well as gene silencing techniques have provided alternative treatment options for these patients.
To find out more about hereditary ATTR amyloidosis visit our dedicated Learning Zone, which contains further information on the neurological and cardiac symptoms.
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Welcome to the Fluid Management Learning Zone. In this Learning Zone we provide an overview of fluid management, including albumin for sepsis and septic shock, guidelines for fluid management in liver cirrhosis and fluid management in cardiac surgery.
Cardiomyopathy is a manifestation of transthyretin amyloidosis (ATTR), which is an underrecognized systemic disease whereby the transthyretin protein misfolds to form fibrils that deposit in various tissues and organs.
Related news and insights
Alnylam Pharmaceuticals presented new results from the Global Open-Label Extension (OLE) study of Onpattro (patisiran), an RNAi therapeutic for the treatment of the polyneuropathy of hereditary ATTR (hATTR) amyloidosis in adults, at the European Academy of Neurology (EAN) Virtual Congress.
Alnylam Pharmaceuticals, Inc. announced that the FDA has granted Fast Track designation to vutrisiran, an investigational therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.
Alnylam Pharmaceuticals, Inc. announced that it has achieved full enrollment in its HELIOS-A Phase III study of vutrisiran an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis. The study was designed to enroll 160 patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy across 68 sites in 22 countries. Alnylam is on track to report topline results from HELIOS-A in early 2021.