Hereditary ATTR Amyloidosis (hATTR)
Hereditary ATTR amyloidosis is a life-threatening disease caused by a mutation in the TTR gene, which results in misfolded TTR that may aggregate to form insoluble amyloid fibrils. These fibrils are deposited in various organs, most commonly accumulating in the nerves, heart and gastrointestinal tract, with heterogeneous presentation and varying prognosis according to the specific genetic mutation.
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Related news and insights
Alnylam Pharmaceuticals, Inc. announced that the HELIOS-A Phase III study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, met its primary and both secondary endpoints at nine months in patients with hATTR amyloidosis with polyneuropathy.
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.
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.