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Transthyretin familial amyloid polyneuropathy
Zhurnal nevrologii i psikhiatrii im. S.S. Korsakova, 2018Transthyretin family amyloid polyneuropathy (TTR-FAP) is a progressive, ultimately fatal disease. It manifests itself primarily with sensory, motor and autonomic polyneuropathy and/or cardiomyopathy and is caused by extracellular deposition of insoluble amyloid fibrils in the endoneurium.
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Pathogenesis of transthyretin amyloidosis
Amyloid, 2012Current dogma for transthyretin (TTR) pathogenesis is that mutations in TTR alter its structure such that the tetramer becomes unstable and prone to release of monomer which then becomes the putative building block of the fibril. This hypothesis is supported by thermodynamic data showing decreased stability of mutant TTR tetrameric proteins and ...
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Tafamidis for transthyretin amyloidosis
Drugs of Today, 2012Tafamidis meglumine (Vyndaqel®, Pfizer) is a novel, first-in-class drug for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP), a rare neurodegenerative disorder characterized by progressive sensory, motor and autonomic impairment that is ultimately fatal.
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CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
New England Journal of Medicine, 2021Julian D Gillmore +2 more
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La Revue de medecine interne, 2007
Transthyretin amyloidoses are the most common form of amyloidosis. Two different types of transthyretin amyloidoses are described, one is rare, familial, its precursor is the mutated transthyretin, this type is called transthyretin amyloid, the other is more common, its precursor is wild transthyretin, this second type is called senile amyloid.
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Transthyretin amyloidoses are the most common form of amyloidosis. Two different types of transthyretin amyloidoses are described, one is rare, familial, its precursor is the mutated transthyretin, this type is called transthyretin amyloid, the other is more common, its precursor is wild transthyretin, this second type is called senile amyloid.
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Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid
New England Journal of Medicine, 2023Fabian aus dem Siepen +2 more
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Isolated transthyretin valvular amyloidosis
European Heart Journal, 2023Yi-Tsang Fu, Ling Kuo, Wen-Chung Yu
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Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis
New England Journal of Medicine, 2023Mathew S Maurer +2 more
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Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy
New England Journal of Medicine, 2018Perry M Elliott +2 more
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Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
New England Journal of Medicine, 2018John L Berk +2 more
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