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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Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed AstraZeneca and Ionis’ eplontersen met its co-primary endpoints in a planned interim analysis at 35 weeks.
Alnylam announces 3-month extension of review period for new drug application for vutrisiran to treat ATTR amyloidosis. Alnylam Pharmaceuticals, Inc., a RNAi therapeutics company, announced that the FDA has extended the review timeline of the New Drug Application (NDA) for vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, to allow for the review of newly added information related to the new secondary packaging and labelling facility.
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.