Disease page
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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