ATTR amyloidosis


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Related to ATTR amyloidosis: Hereditary amyloidosis, familial amyloidosis

ATTR amyloidosis

the most common form of familial amyloidosis, in which any of numerous mutations of the gene encoding transthyretin cause systemic autosomal-dominant disorders characterized by polyneuropathies, cardiomyopathies, and variable organ involvement.
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This restructuring provides Alnylam with the opportunity to consolidate its ATTR amyloidosis business to maximize its value, and the opportunity for near-term acceleration of product revenue growth based on newly obtained rights to commercialize patisiran around the world, once approved.
Full results will be presented at the First European ATTR Amyloidosis Meeting for Patients and Doctors on 2 November, in Paris, France.
6-8) The most common association is systemic AL amyloidosis, (9,10) but cases of diffuse alveolar-septal amyloidosis that are caused by systemic AA, systemic wild-type ATTR, and systemic hereditary ATTR amyloidosis have also been reported (Table 2).
Patisiran, an investigational intravenously administered RNAi therapeutic targeting transthyretin in development for the treatment of hereditary ATTR amyloidosis, is designed to silence specific messenger RNA, potentially blocking the production of TTR protein.